Log In

Forgot Password?

OR

Not a member? Continue as a nonmember.

Become a Member

By becoming a member of the AAN, you can receive exclusive information to help you at every stage of your career. Benefits include:

Join Now See All Benefits

Loading... please wait

2022 Annual Meeting | S39 - Child Neurology and Developmental Neurology

Thursday 04/07/22
03:30 PM - 05:30 PM PDT Add To Calendar
This program will be presented both in-person and online
This program will be available in the meeting's On Demand product.
Scientific Platform Session
Child Neurology and Developmental Neurology
2.00 CME credits

Program Materials Program Evaluations

Event Timeline
03:30 PM - 03:30 PM PDT Speaker Walk-in Slides
Erin Jackson
03:30 PM - 03:42 PM PDT Abstract 001 - Development of Therapy for Canavan Disease Using Human Induced Pluripotent Stem Cells
Neal Prakash, MD, PhD, FAAN
03:42 PM - 03:54 PM PDT Abstract 002 - Effectiveness and Safety of Onasemnogene Abeparvovec in Older Patients with Spinal Muscular Atrophy (SMA): Real-World Outcomes from the RESTORE Registry
Laurent Servais, MD,PhD
03:54 PM - 04:06 PM PDT Abstract 003 - SUNFISH: 3-year Efficacy and Safety of Risdiplam in Types 2 and 3 SMA
John W. Day, MD, PhD
04:06 PM - 04:18 PM PDT Abstract 004 - Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and Two Copies of SMN2
Kevin Strauss, MD
04:18 PM - 04:30 PM PDT Abstract 005 - FIREFISH Parts 1 and 2: 24-month Safety and Efficacy of Risdiplam in Type 1 SMA
John W. Day, MD, PhD
04:30 PM - 04:42 PM PDT Abstract 006 - Eladocagene Exuparvovec Gene Therapy Improves Motor Development in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency
Paul Wuh-Liang Hwu
04:42 PM - 04:54 PM PDT Abstract 007 - Phase 2/3 Clinical Trial Results: Safety & Efficacy Results of RT001, a site-specific (C11) di-deutero synthetic homologue of linoleic acid, in Infantile Neuroaxonal Dystrophy
Alexander James Fay, MD, PhD
04:54 PM - 05:06 PM PDT Abstract 008 - Updated results of Transpher A, a multicenter, single-dose, Phase 1/2 clinical trial of ABO-102 investigational gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
Kevin M. Flanigan, MD, FAAN
05:06 PM - 05:18 PM PDT Abstract 009 - Contributions of Purine Synthesis and Recycling During Brain Development: Relevance to Lesch Nyhan Disease
Fatemeh Seifar
05:18 PM - 05:30 PM PDT Abstract 010 - Systematic Analysis of Brain MRI Findings in Adaptor Protein Complex 4 – associated Hereditary Spastic Paraplegia Reveals Patterns for Diagnosis and Disease Progression
Darius Ebrahimi-Fakhari, MD, PhD
Faculty Disclosures
Ksenija Gorni Dr. Gorni has received personal compensation for serving as an employee of Hoffman La Roche.
Wai Yin Yeung Wai Yin Yeung has nothing to disclose.
Odile Boespflug-Tanguy Odile Boespflug-Tanguy has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Minoryx. Odile Boespflug-Tanguy has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Inventiva. Odile Boespflug-Tanguy has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for BLUEBIRD.
Carole Vuillerot Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Hoffmann Roche. Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen . Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche Hoffmann. Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avexis.
Giovanni Baranello Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AveXis. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for AveXis. The institution of Giovanni Baranello has received research support from Great Ormond Street Hospital Charity.
Kathryn J. Swoboda, MD Dr. Swoboda has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Swoboda has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. The institution of Dr. Swoboda has received research support from Biogen.
Jena M. Krueger, MD Dr. Krueger has nothing to disclose.
Afshin Saffari, MD Dr. Saffari has nothing to disclose.
Craig M. Zaidman, MD Dr. Zaidman has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for sarepta. The institution of Dr. Zaidman has received research support from Washington University in St Louis. The institution of Dr. Zaidman has received research support from Novartis.
Diane J. Sutcliffe, Other Ms. Sutcliffe has nothing to disclose.
Deepa Chand Deepa Chand has received personal compensation for serving as an employee of Novartis Gene Therapies. Deepa Chand has received stock or an ownership interest from Novartis. Deepa Chand has received publishing royalties from a publication relating to health care.
Francesco Muntoni, MD Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Avexis. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. The institution of Dr. Muntoni has received research support from European Commission. The institution of Dr. Muntoni has received research support from Medical Research Council. The institution of Dr. Muntoni has received research support from Biogen. The institution of Dr. Muntoni has received research support from Muscular Dystrophy UK. The institution of Dr. Muntoni has received research support from MDA USA. The institution of Dr. Muntoni has received research support from Sarepta. The institution of Dr. Muntoni has received research support from Association Francoise Myopathies. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving as a Clinical expert with UK NICE Committee.
Maria Mazurkiewicz-Beldzin Dr. Mazurkiewicz Beldzin has received personal compensation in the range of $0-$499 for serving as a Consultant for Biogen, . Dr. Mazurkiewicz Beldzin has received personal compensation in the range of $0-$499 for serving as a Consultant for Novartis. Dr. Mazurkiewicz Beldzin has received personal compensation in the range of $0-$499 for serving as a Consultant for Roche. Dr. Mazurkiewicz Beldzin has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Mazurkiewicz Beldzin has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis.
Bryan McGill, MD, PhD Dr. McGill has received personal compensation for serving as an employee of Novartis. Dr. McGill has received personal compensation for serving as an employee of AveXis. Dr. McGill has received stock or an ownership interest from Novartis.
Marianne Gerber Marianne Gerber has received personal compensation for serving as an employee of Roche. Marianne Gerber has received stock or an ownership interest from F Hoffmann La Roche .
Laurent Servais, MD,PhD The institution of Dr. Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. The institution of Dr. Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. The institution of Dr. Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Avexis. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dynacure. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Audentes. The institution of Dr. Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Pfizer. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Santhera. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for RegenexBio. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Affinia. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Anagenesis. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Catabasis. The institution of Dr. Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Evox. Dr. Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Lupin. Dr. Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fibrogen.
Mustafa Sahin, MD, PhD Dr. Sahin has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Regenxbio . Dr. Sahin has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Centene. Dr. Sahin has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Celgene. Dr. Sahin has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for PTEN Research. Dr. Sahin has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. The institution of Dr. Sahin has received research support from NIH. The institution of Dr. Sahin has received research support from Tuberous Sclerosis Alliance. The institution of Dr. Sahin has received research support from Charities Aid Foundation. The institution of Dr. Sahin has received research support from Simons Foundation Autism Research Initiative. The institution of Dr. Sahin has received research support from Aucta Pharmaceuticals . The institution of Dr. Sahin has received research support from Loulou Foundation.
Lizhao Feng, PhD Dr. Feng has nothing to disclose.
Kevin Strauss, MD Dr. Strauss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis Gene Therapies. Dr. Strauss has received research support from Novartis Gene Therapies.
Kayoko Saito, MD, PhD Dr. Saito has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapies, Inc./Novartis. Dr. Saito has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Saito has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai (Roche). Dr. Saito has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Novartis Gene Therapies, Inc./Novartis. Dr. Saito has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Chugai (Roche). The institution of Dr. Saito has received research support from Biogen.
Mark Midei, MD Dr. Midei has received personal compensation for serving as an employee of Retrotope. Dr. Midei has received stock or an ownership interest from Retrotope.
Eduardo Tizzano Eduardo Tizzano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Eduardo Tizzano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avexis Novartis.
Nathalie M. Goemans, MD Dr. Goemans has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Goemans has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Dr. Goemans has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Dr. Goemans has received publishing royalties from a publication relating to health care.
Frederic Heerinckx, PharmD No disclosure on file
Omar Dabbous Omar Dabbous has received personal compensation for serving as an employee of Novartis Gene Therapies. Omar Dabbous has received stock or an ownership interest from Novartis Gene Therapies.
Michelle A. Farrar Michelle A. Farrar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Michelle A. Farrar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Michelle A. Farrar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Michelle A. Farrar has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen. Michelle A. Farrar has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche.
Jianfei Chao, PhD Dr. Chao has received personal compensation for serving as an employee of City of Hope.
Darius Ebrahimi-Fakhari, MD, PhD Dr. Ebrahimi-Fakhari has received personal compensation in the range of $0-$499 for serving as a Consultant for Alcimed Inc.. The institution of Dr. Ebrahimi-Fakhari has received research support from CureAP4 Foundation, CureSPG50 Foundation, Spastic Paraplegia Foundation, Manton Center for Orphan Disease Research, BPAN Warriors Foundation, NIH/NINDS. Dr. Ebrahimi-Fakhari has received publishing royalties from a publication relating to health care.
Dmitry Vlodavets Dmitry Vlodavets has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Dmitry Vlodavets has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche . Dmitry Vlodavets has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dmitry Vlodavets has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dmitry Vlodavets has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Novartis. Dmitry Vlodavets has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Roche. Dmitry Vlodavets has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for PTC Therapeutics. Dmitry Vlodavets has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Dmitry Vlodavets has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for PTC Therapeutics. Dmitry Vlodavets has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche.
Darryl C. De Vivo, MD, FAAN Dr. De Vivo has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen and Novartis. Dr. De Vivo has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aspa Therapeutics.
Alexander James Fay, MD, PhD Dr. Fay has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Retrotope. Dr. Fay has received personal compensation in the range of $0-$499 for serving as a Consultant for Teladoc. Dr. Fay has received personal compensation in the range of $0-$499 for serving as a Consultant for Guidepoint. Dr. Fay has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Aeglea. Dr. Fay has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis . Dr. Fay has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Curry Rockefeller Group. Dr. Fay has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aspiro. Dr. Fay has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Dwyer Hernandez. Dr. Fay has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Gallagher Bassett. Dr. Fay has received research support from Sarepta. The institution of Dr. Fay has received research support from Preston-Werner Ventures.
ML Couce ML Couce has nothing to disclose.
Piyush Joshi, PhD Dr. Joshi has nothing to disclose.
Ana Del Campo, PhD Ms. Del Campo has received personal compensation for serving as an employee of Abeona Therapeutics. Ms. Del Campo has received stock or an ownership interest from Abeona Therapeutics.
Ricardo Masson Ricardo Masson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche, Biogen, Avexis. Ricardo Masson has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche, Biogen, Avexis.
Yanhong Shi, MD Dr. Kyei-Frimpong has nothing to disclose.
Carmen Martin Carmen Martin has received personal compensation for serving as an employee of Roche Products Ltd. Carmen Martin has received stock or an ownership interest from F. Hoffmann-La Roche Ltd.
Andres Martinez-Porras MJ de Castro has nothing to disclose.
Peter Milner Peter Milner has received personal compensation for serving as an employee of Retrotope . Peter Milner has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for Retrotope . Peter Milner has received stock or an ownership interest from Retrotope .
Hui Xiong Hui Xiong has nothing to disclose.
Melissa Menier Melissa Menier has received personal compensation for serving as an employee of ATLAS Clarity, LLC.
Angela Dodman Angela Dodman has received personal compensation for serving as an employee of F. Hoffman-La Roche Ltd.
Hugh John McMillan, MD Dr. McMillan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis Gene Therapies. Dr. McMillan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis Gene Therapies. The institution of Dr. McMillan has received research support from Roche.
Anna *USE ID 361430 Kostera-Pruszczyk Anna Kostera-Pruszczyk has nothing to disclose.
Jerry R. Mendell, MD, FAAN Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vertex. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics . The institution of Dr. Mendell has received research support from Sarepta.
Edmar Zanoteli Edmar Zanoteli has nothing to disclose.
Maryam Oskoui, MD, FAAN Dr. Oskoui has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for the Association des Neurologues du Quebec. The institution of Dr. Oskoui has received research support from Roche Genetech. The institution of Dr. Oskoui has received research support from Muscular Dystrophy Canada. The institution of Dr. Oskoui has received research support from Canadian Institutes of Health Research. The institution of Dr. Oskoui has received research support from Santhera. Dr. Oskoui has a non-compensated relationship as a Member of the Medical and Scientific Advisory Committee with Muscular Dystrophy Canada that is relevant to AAN interests or activities.
Igor Grachev No disclosure on file
Heidemarie Kletzl Heidemarie Kletzl has received personal compensation for serving as an employee of Hoffmann-La Roche.
Perry Shieh, MD, PhD, FAAN Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for CSL Behring. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Grifols. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Shieh has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Biogen. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Genentech. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Catalyst.
Andrea Klein Andrea Klein has nothing to disclose.
Julian Alecu, Other Mr. Alecu has nothing to disclose.
Fred Anderson, PhD Dr. Anderson has nothing to disclose.
Elena Mazzone Elena Mazzone has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Avexis. Elena Mazzone has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Elena Mazzone has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Biogen.
Nicholas JC Smith Nicholas JC Smith has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. The institution of Nicholas JC Smith has received research support from Medical Research Futures Fund - Australia. The institution of Nicholas JC Smith has received research support from Sanfilippo Children's Foundation. The institution of Nicholas JC Smith has received research support from Abeona Therapeutics. The institution of Nicholas JC Smith has received research support from Bluebird Bio.
Kristy Rose Kristy Rose has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Roche. Kristy Rose has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Kristy Rose has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Roche. Kristy Rose has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen.
Fatemeh Seifar Ms. Seifar has nothing to disclose.
Traci B. Schilling, MD No disclosure on file
Nicholas Deconinck Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving as a Consultant for biogen. Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for roche. Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for avexis. Nicholas Deconinck has received publishing royalties from a publication relating to health care.
Astrid Pañeda, PhD Ms. Pañeda has received personal compensation for serving as an employee of Abeona Therapeutics. Ms. Pañeda has received personal compensation for serving as an employee of Forge Biologics . Ms. Pañeda has received stock or an ownership interest from Abeona Therapeutics Inc.
Sanjay Prabhu Sanjay Prabhu has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for National Medical Consultants. Sanjay Prabhu has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Various law firms.
Kevin M. Flanigan, MD, FAAN Dr. Flanigan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Apic Bio. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AavantiBio. Dr. Flanigan has stock in 4D Molecular Therapeutics. The institution of Dr. Flanigan has received research support from Abeona Therapeutics. The institution of Dr. Flanigan has received research support from Sarepta Therapeutics. The institution of Dr. Flanigan has received research support from Astellas Therapeutics. Dr. Flanigan has received intellectual property interests from a discovery or technology relating to health care.
KV Truxal The institution of KV Truxal has received research support from Abeona Therapeutics.
Sitra Tauscher-Wisniewski Sitra Tauscher-Wisniewski has received personal compensation for serving as an employee of Novartis Gene Therapies.
Marvin Ziegler Mr. Ziegler has received research support from German National Academic Foundation. Mr. Ziegler has received research support from German Academic Exchange Service.
Edward Yang, MD,PhD The institution of Dr. Yang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Syngeos.
Maria Escolar Maria Escolar has received personal compensation for serving as an employee of Forge Biologics. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Azafaros. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for JCR. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Guidepoint. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for BridgeBio. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for TEGA Therapeutics. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Orphazyme. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Shire/Takeda. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aeglea Bio. Maria Escolar has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Shire/Takeda. Maria Escolar has received stock or an ownership interest from Forge Biologics. The institution of an immediate family member of Maria Escolar has received research support from The Legacy of Angels. The institution of an immediate family member of Maria Escolar has received research support from NIH. The institution of Maria Escolar has received research support from The Legacy of Angels. Maria Escolar has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Maria Escolar has received intellectual property interests from a discovery or technology relating to health care.
Susana Quijano-Roy, MD,PhD Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis GT. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SAnofi Genzyme. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AVEXIS. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ROCHE. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NOVARTIS GT. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Prof. Quijano-Roy has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis GT. Prof. Quijano-Roy has received publishing royalties from a publication relating to health care.
Julie Anne Parsons, MD Dr. Parsons has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Parsons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Parsons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Parsons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ultragenex. The institution of Dr. Parsons has received research support from AveXis. The institution of Dr. Parsons has received research support from Biogen. The institution of Dr. Parsons has received research support from Scholar Rock. The institution of Dr. Parsons has received research support from Biohaven. The institution of Dr. Parsons has received research support from PTC Therapeutics. The institution of Dr. Parsons has received research support from Novartis.
J Ruiz J Ruiz has received personal compensation for serving as an employee of Abeona Therapeutics. J Ruiz has received stock or an ownership interest from Abeona Therapeutics.
Basil T. Darras, MD The institution of Dr. Darras has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Darras has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus. The institution of Dr. Darras has received research support from National Institutes of Health/National Institute of Neurological Disorders and Stroke,. The institution of Dr. Darras has received research support from Slaney Family Fund for SMA. The institution of Dr. Darras has received research support from Spinal Muscular Atrophy Foundation. The institution of Dr. Darras has received research support from CureSMA. The institution of Dr. Darras has received research support from Working on Walking Fund . The institution of Dr. Darras has received research support from CHERISH, CS2/CS12 . The institution of Dr. Darras has received research support from Biogen for CS11. The institution of Dr. Darras has received research support from AveXis. The institution of Dr. Darras has received research support from Sarepta Pharmaceuticals. The institution of Dr. Darras has received research support from PTC Therapeutics. The institution of Dr. Darras has received research support from Roche. The institution of Dr. Darras has received research support from Santhera. The institution of Dr. Darras has received research support from Scholar Rock. The institution of Dr. Darras has received research support from Fibrogen. The institution of Dr. Darras has received research support from Summit. Dr. Darras has received publishing royalties from a publication relating to health care. Dr. Darras has received publishing royalties from a publication relating to health care.
Thomas Macek Thomas Macek has received personal compensation for serving as an employee of Novartis . Thomas Macek has received stock or an ownership interest from Novartis .
Yin-Hsiu Chien Yin-Hsiu Chien has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi Genzyme. Yin-Hsiu Chien has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Yin-Hsiu Chien has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Yin-Hsiu Chien has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Yin-Hsiu Chien has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Yin-Hsiu Chien has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sanofi Genzyme. Yin-Hsiu Chien has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Avexis/Norvatis. Yin-Hsiu Chien has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. The institution of Yin-Hsiu Chien has received research support from Sanofi Genzyme.
Susan T. Iannaccone, MD, FAAN Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AveXis. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Iannaccone has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. The institution of Dr. Iannaccone has received research support from AveXis. The institution of Dr. Iannaccone has received research support from Biogen. The institution of Dr. Iannaccone has received research support from Sarepta. The institution of Dr. Iannaccone has received research support from PTC Therapeutics. The institution of Dr. Iannaccone has received research support from FibroGen. The institution of Dr. Iannaccone has received research support from ReveraGen. The institution of Dr. Iannaccone has received research support from MDA. The institution of Dr. Iannaccone has received research support from PPMD. The institution of Dr. Iannaccone has received research support from NIH. Dr. Iannaccone has received personal compensation in the range of $0-$499 for serving as a grant reviewer with NIH.
Darius Adams Darius Adams has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Retrotope .
John W. Day, MD, PhD Dr. Day has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Affinia Therapeutics. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapy. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche/Genentech. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for Muscular Dystrophy Association. The institution of Dr. Day has received research support from Audentes. The institution of Dr. Day has received research support from Novartis Gene Therapy. The institution of Dr. Day has received research support from Biogen. The institution of Dr. Day has received research support from Cytokinetics. The institution of Dr. Day has received research support from Roche/Genentech. The institution of Dr. Day has received research support from Sanofi/Genzyme. The institution of Dr. Day has received research support from Sarepta. The institution of Dr. Day has received research support from Scholar Rock. Dr. Day has received intellectual property interests from a discovery or technology relating to health care.
Renata Scalco Renata SCALCO has received personal compensation for serving as an employee of Roche. Renata SCALCO has received stock or an ownership interest from Roche.
Laurent Servais Laurent Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Laurent Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. The institution of Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Evox. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sysnav. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BioHaven. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zentech. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MitoRx. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Lupin. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fibrogen. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alltrana. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Illumina. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Laurent Servais has received research support from Roche. The institution of Laurent Servais has received research support from Novartis. The institution of Laurent Servais has received research support from Biogen. The institution of Laurent Servais has received research support from Zentech. The institution of Laurent Servais has received research support from BioHaven. The institution of Laurent Servais has received research support from PerkinHalmers. The institution of Laurent Servais has received research support from Scholar Rock.
Chun-Hwei Tai The institution of Chun-Hwei Tai has received research support from National Taiwan University Hospital.
H. A. Jinnah, MD, PhD, FAAN Dr. Jinnah has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takaha/Ene. Dr. Jinnah has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Ipsen. Dr. Jinnah has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ipsen. Dr. Jinnah has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abbvie. The institution of Dr. Jinnah has received research support from Addex. The institution of Dr. Jinnah has received research support from Aeon. The institution of Dr. Jinnah has received research support from Revance. The institution of Dr. Jinnah has received research support from Jazz.
Andrés Nascimento, MD Dr. Nascimento has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche, Novartis , Biogen. Dr. Nascimento has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche, Novartis, Biogen.
Eugenio Mercuri Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis, Scholar Rock, Cytokinetics. Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis. The institution of Eugenio Mercuri has received research support from biogen.
Jessica Braid, Other Miss Braid has received stock or an ownership interest from Roche Products Ltd.
Sarah Kavanagh Sarah Kavanagh has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for UCB Pharma. Sarah Kavanagh has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Colorado Prevention Center. Sarah Kavanagh has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for PureTech Health. Sarah Kavanagh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Worldwide Clinical Trails.
Jennifer M. Kwon, MD, FAAN Dr. Kwon has nothing to disclose.
Richard S. Finkel, MD, FAAN Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Capricor. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ReveraGen. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. The institution of Dr. Finkel has received research support from AveXis. The institution of Dr. Finkel has received research support from Biogen. The institution of Dr. Finkel has received research support from Capricor. The institution of Dr. Finkel has received research support from Catabasis. The institution of Dr. Finkel has received research support from ReveraGen. The institution of Dr. Finkel has received research support from Roche. The institution of Dr. Finkel has received research support from Scholar Rock. Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. Dr. Finkel has received personal compensation in the range of $0-$499 for serving as a Speaker in workshop with National Academy of Sciences. Dr. Finkel has a non-compensated relationship as a advisor with n-Lorem Foundation that is relevant to AAN interests or activities. Dr. Finkel has a non-compensated relationship as a Board Member with EveryLife Foundation that is relevant to AAN interests or activities.
Antonia Wang, PhD Dr. Wang has received personal compensation for serving as an employee of PTC Therapeutics. Dr. Wang has received stock or an ownership interest from PTC Therapeutics, Inc..
Ni-Chung Lee Ni-Chung Lee has nothing to disclose.
Paldeep Atwal Paldeep Atwal has received personal compensation for serving as an employee of Retrotope.
Nicole *USE ID 202625 LaMarca Nicole Marie LaMarca has received personal compensation for serving as an employee of Novartis Gene Therapies.
KL McBride The institution of KL McBride has received research support from Abeona. The institution of KL McBride has received research support from Homology Medicine Inc. The institution of KL McBride has received research support from Biomarin.
Muna El-Khairi Muna El-Khairi has received personal compensation for serving as an employee of Roche Products Limited.
Janbernd Kirschner Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genethon. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Italfarmaco. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC. The institution of Janbernd Kirschner has received research support from Roche. The institution of Janbernd Kirschner has received research support from Biogen. The institution of Janbernd Kirschner has received research support from Novartis.
Claudia A. Chiriboga, MD, FAAN Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentec. Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Dr. Chiriboga has received research support from Roche. The institution of Dr. Chiriboga has received research support from Avexis/Novartis. The institution of Dr. Chiriboga has received research support from Biogen. The institution of Dr. Chiriboga has received research support from NIH. The institution of Dr. Chiriboga has received research support from Biohaven. The institution of Dr. Chiriboga has received research support from Genentec. Dr. Chiriboga has received publishing royalties from a publication relating to health care.
Eleni Gaki, MD Dr. Gaki has received personal compensation for serving as an employee of Roche. Dr. Gaki has received stock or an ownership interest from Roche Products Ltd.
Sheng-Hong Tseng Sheng-Hong Tseng has nothing to disclose.
M Fuller The institution of M Fuller has received research support from Sanofi. The institution of M Fuller has received research support from Takeda. The institution of M Fuller has received research support from Abeona.
Neal Prakash, MD, PhD, FAAN Dr. Prakash has nothing to disclose.
Bhooma Rajagopalan Aravamuthan, MD, PhD Dr. Aravamuthan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurocrine Biosciences. An immediate family member of Dr. Aravamuthan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for SK Life Science, Inc.. Dr. Aravamuthan has received research support from National Institute of Neurological Disorders and Stroke. Dr. Aravamuthan has received publishing royalties from a publication relating to health care.
Paul Wuh-Liang Hwu Paul Wuh-Liang Hwu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics Inc.. Paul Wuh-Liang Hwu has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC therapeutics. The institution of Paul Wuh-Liang Hwu has received research support from PTC therapeutics.
Alison L. Christy, MD, PhD Dr. Christy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Azurity. Dr. Christy has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for SAGE Publishing. Dr. Christy has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MDLinx. Dr. Christy has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MDLinx. The institution of Dr. Christy has received research support from Biohaven. The institution of Dr. Christy has received research support from Novartis / Amgen. The institution of Dr. Christy has received research support from Eli Lilly. The institution of Dr. Christy has received research support from Abbvie. The institution of Dr. Christy has received research support from Eli Lilly.
Erin Jackson Ms. Jackson has nothing to disclose.