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2023 Annual Meeting | S34 - Child Neurology and Developmental Neurology 2

Wednesday 04/26/23
01:00 PM - 03:00 PM EST 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
A group of abstracts covering a similar topic presented in an oral format. Presentations are eight minutes in length with four minutes for question and answer with the author.
2.00 CME credits
Fellow, Resident, General Neurologist, Specialist Neurologist, Non-neurologist, Advanced Practice Provider, Neurohospitalist, Medical Student
Didactic

Program Materials Program Evaluations

Event Timeline
01:00 PM - 01:12 PM EST Abstract 001 - The Natural History of Motor, Cognitive Development and Mood in Early Onset Hereditary Spastic Paraplegias
Michelle Rose Christie, MD
01:12 PM - 01:24 PM EST Abstract 002 - Intravenous and Intrathecal Onasemnogene Abeparvovec Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy: Long-Term Follow-Up Study
Basil T. Darras, MD
01:24 PM - 01:36 PM EST Abstract 003 - Immunotherapy Responsiveness and Risk of Relapse in Down Syndrome Regression Disorder
Jonathan Santoro, MD
01:36 PM - 01:48 PM EST Abstract 004 - Eladocagene Exuparvovec Gene Therapy Improves Motor Development in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency
Alexis Russell
01:48 PM - 02:00 PM EST Abstract 005 - Skin-dependent morphological and molecular maturation of specialized mechanosensory neurons
Charalampia Koutsioumpa, MD, PhD
02:00 PM - 02:12 PM EST Abstract 006 - Pediatric Neuroinflammatory Disease in the Era of COVID-19: Single Center Survey of the Medical and Social Impact of COVID-19 and COVID-19 Vaccines
Mara Bahri
02:12 PM - 02:24 PM EST Abstract 007 - PRAX-562 is a Well-Tolerated, Novel Persistent Sodium Channel Blocker with Broad Anticonvulsant Activity in Multiple DEE Mouse Models
Lyndsey Anderson Anderson, PhD
02:24 PM - 02:36 PM EST Abstract 008 - Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry
Vinay Penematsa
02:36 PM - 02:48 PM EST Abstract 009 - SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA)
Laurent Servais
02:48 PM - 03:00 PM EST Abstract 010 - Evaluation of the Biodistribution, Efficacy, and Side-Effect Profile of Deflazacort, Prednisolone and Vamorolone in a DMD Mouse Model
Grace Liu, PhD
Faculty Disclosures
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.
Anne M. Connolly, MD, FAAN Dr. Connolly has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Edgewise Therapeutics. Dr. Connolly has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Octapharma. Dr. Connolly has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Connolly has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Biohaven. Dr. Connolly 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. Connolly has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Morphic 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.
Kazuhiro Haginoya, MD Dr. Haginoya has nothing to disclose.
Robyn Ann Filipink, MD The institution of an immediate family member of Dr. Filipink has received research support from NIH . Dr. Filipink has received personal compensation in the range of $0-$499 for serving as a Author with Medlink.
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 Biogen. 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. 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. Dr. Oskoui has a non-compensated relationship as a Member of the External Advisory Board with MDUK Oxford Neuromuscular Centre that is relevant to AAN interests or activities.
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.
Michael S. Rafii, MD, PhD Dr. Rafii has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AC Immune. Dr. Rafii has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Keystone Bio. Dr. Rafii has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alzheon.
Leigh Ramos-Platt, MD Dr. Ramos-Platt has received personal compensation for serving as an employee of MDA. Dr. Ramos-Platt has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint. The institution of Dr. Ramos-Platt has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Ramos-Platt has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cure sma.
Jean-Baptiste Le Pichon, MD, PhD Dr. Le Pichon has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Every case is with a different law firm.. The institution of Dr. Le Pichon has received research support from Health Resources and Health Services Administration (HRSA).
Lauren Morgenroth Ms. Morgenroth has received personal compensation for serving as an employee of TRiNDS. Ms. Morgenroth has stock in TRiNDS.
Craig McDonald, MD Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for PTC Therapeutics. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Dr. McDonald has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Capricor Therapeutics. Dr. McDonald has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Catabasis. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epirium Bio. Dr. McDonald has received personal compensation in the range of $0-$499 for serving as a Consultant for Italfarmaco. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Santhera Pharmaceuticals. Dr. McDonald 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 Therapeutics. Dr. McDonald 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. Dr. McDonald 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. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis Pharmaceuticals. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Santhera Pharmaceuticals. The institution of Dr. McDonald has received research support from Sarepta Therapeutics. The institution of Dr. McDonald has received research support from PTC Therapeutics. The institution of Dr. McDonald has received research support from Santhera Pharmaceuticals. The institution of Dr. McDonald has received research support from Astellas. The institution of Dr. McDonald has received research support from Capricor Therapeutics. The institution of Dr. McDonald has received research support from Catabasis. The institution of Dr. McDonald has received research support from Epirium Bio. The institution of Dr. McDonald has received research support from Italfarmaco. Dr. McDonald has received research support from NS Pharma. The institution of Dr. McDonald has received research support from Pfizer. The institution of Dr. McDonald has received research support from NIH (NINDS). The institution of Dr. McDonald has received research support from NIDILRR. The institution of Dr. McDonald has received research support from Department of Defense. The institution of Dr. McDonald has received research support from Parent Project Muscular Dystrophy. The institution of Dr. McDonald has received research support from Muscular Dystrophy Association. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Member National Advisory Board for Medical Rehabilitation Research with NIH.
Ksenija Gorni Dr. Gorni has received personal compensation for serving as an employee of Hoffman La Roche.
Ellen Welch Ms. Welch has received personal compensation for serving as an employee of PTC Therapeutics. Ms. Welch has stock in PTC Therapeutics.
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.
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.
George Capone The institution of George Capone has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ACImmune. The institution of George Capone has received research support from LuMind.
Grace Gombolay, MD Dr. Gombolay has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Pediatric Neurology. An immediate family member of Dr. Gombolay has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Washington Injury Lawyers. The institution of Dr. Gombolay has received research support from CDC. The institution of Dr. Gombolay has received research support from NIH.
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.
Anna *USE ID 361430 Kostera-Pruszczyk Anna Kostera-Pruszczyk has nothing to disclose.
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.
Heather Gordish-Dressman Heather Gordish-Dressman has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AGADA BioSciences. Heather Gordish-Dressman has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for TRiNDS, LLC. Heather Gordish-Dressman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Rejuvenate Bio. An immediate family member of Heather Gordish-Dressman has received intellectual property interests from a discovery or technology relating to health care. Heather Gordish-Dressman has received personal compensation in the range of $500-$4,999 for serving as a TACT Advisory Board Reviewer with TREAT-NMD.
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.
Jonathan Santoro, MD Dr. Santoro has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Santoro has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cycle Pharma.
Efthimia Leonardi Efthimia Leonardi has received personal compensation for serving as an employee of PTC Therapeutics, Inc. Efthimia Leonardi has stock in {TC Therapeutics Inc.
Melissa Hutchinson, MD Dr. Hutchinson has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Katz, Korin, Cunningham Attorneys At Law.
Rafael Sierra, PhD Dr. Sierra has received personal compensation for serving as an employee of PTC Therapeutics Inc. Dr. Sierra has stock in PTC Therapeutics Inc.
Michelle Rose Christie, MD Dr. Christie has nothing to disclose.
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.
Panayiota Trifillis Panayiota Trifillis has received personal compensation for serving as an employee of PTC Therapeutics. Panayiota Trifillis has received stock or an ownership interest from PTC Therapeutics, Inc.. Panayiota Trifillis has received intellectual property interests from a discovery or technology relating to health care.
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.
Christian Werner Christian Werner has received personal compensation for serving as an employee of PTC Therapeutics .
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.
Maria Bernadete Resende No disclosure on file
Samuel Mackenzie, MD, PhD Dr. Mackenzie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Phlow, Corp. Dr. Mackenzie has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Everyday Health, Inc. Dr. Mackenzie has received intellectual property interests from a discovery or technology relating to health care.
Ryan Kammeyer, MD The institution of Dr. Kammeyer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Kammeyer has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Ogborn-Mihm Trial Lawyers. The institution of Dr. Kammeyer has received research support from Rocky Mountain Multiple Sclerosis Center.
Connie Chou Connie Chou has stock in PTC Therapeutics.
Chun-Hwei Tai The institution of Chun-Hwei Tai has received research support from National Taiwan University Hospital.
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.
Ni-Chung Lee Ni-Chung Lee has nothing to disclose.
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.
Nikola Jancovski Nikola Jancovski has nothing to disclose.
Snezana Maljevic Snezana Maljevic has nothing to disclose.
Christopher Reid Christopher Reid has nothing to disclose.
Steven Petrou Steven Petrou has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Praxis Precision Medicines. Steven Petrou has stock in Praxis Precision Medicines. The institution of Steven Petrou has received research support from Praxis Precision Medicines. The institution of Steven Petrou has received research support from Medical Research Future Fund. Steven Petrou has received intellectual property interests from a discovery or technology relating to health care. Steven Petrou has received intellectual property interests from a discovery or technology relating to health care.
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.
Vinay Penematsa Vinay Penematsa has nothing to disclose.
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.
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.
Wai Yin Yeung Wai Yin Yeung has nothing to disclose.
Kevin Strauss Kevin Strauss has received personal compensation in the range of $500-$4,999 for serving as a Consultant 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.
Lesa Mehl Lesa Mehl has received personal compensation for serving as an employee of AveXis / Novartis Gene Therapies / Novartis Institutes for Biomedical Research, Inc. Lesa Mehl has stock in Novartis.
Mara Bahri Ms. Bahri has nothing to disclose.
Nick Mastrandrea Nick Mastrandrea has received personal compensation for serving as an employee of PTC Therapeutics. Nick Mastrandrea has stock in PTC Therapeutics.
Mellad Khoshnood, MD Dr. Khoshnood 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.
Saba Jafarpour, MD Dr. Jafarpour has nothing to disclose.
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.
Lauren Hurst Ms. Hurst has nothing to disclose.
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.
Jessica Braid, Other Miss Braid has received stock or an ownership interest from Roche Products Ltd.
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..
Melanie Manning, MD Dr. Manning has nothing to disclose.
Jonathan Blaize, PhD Dr. Blaize has stock in PTC Therapeutics Inc.
Elaine Dong, Other Dr. Dong has nothing to disclose.
Charalampia Koutsioumpa, MD, PhD Dr. Koutsioumpa has nothing to disclose.
Heather Van Mater, MD Dr. Van Mater has nothing to disclose.
Lina Patel, Other Dr. Patel has nothing to disclose.
Ruth C. Brown, PhD Dr. Brown has nothing to disclose.
Panteha Hayati Rezvan, PhD Dr. Hayati Rezvan has nothing to disclose.
Noemi A. Spinazzi, MD Dr. Spinazzi has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Zalkin Law Firm. The institution of Dr. Spinazzi has received research support from Konica Minolta.
Gordon Worley, MD Dr. Worley has nothing to disclose.
Luke Dwyer, MD Dr. Dwyer has nothing to disclose.
Natalie K. Boyd Ms. Boyd has nothing to disclose.
Maria Del Carmen Ortega Bernardo, Sr., MD Dr. ORTEGA BERNARDO has nothing to disclose.
Rebecca Partridge, MD Dr. Partridge has nothing to disclose.
Elise Michelle Sannar, MD The institution of Dr. Sannar has received research support from Colorado Department of Public Health and Environment. The institution of Dr. Sannar has received research support from National Institute of Health.
Abhik Banerjee, PhD Dr. Banerjee has nothing to disclose.
Maria A. Stanley, MD The institution of Dr. Stanley has received research support from NIH. The institution of Dr. Stanley has received research support from NIH.
Eileen A. Quinn, MD Dr. Quinn has nothing to disclose.
Shirley Yeh Mrs. Yeh has nothing to disclose.
Min Lin, PhD Dr. Lin has received personal compensation for serving as an employee of PTC Therapeutics.
Shelley Yvonne Johnson, Other Ms. Johnson has received personal compensation for serving as an employee of PTC Therapeutics Inc. Ms. Johnson has stock in PTC Therapeutics.
Lanqing Ying, PhD Ms. Ying has received personal compensation for serving as an employee of PTC Therapeutics.
Jana Narasimhan, PhD Dr. Narasimhan has received personal compensation for serving as an employee of PTC Therapeutics Inc. Dr. Narasimhan has stock in PTC Therapeutics Inc. Dr. Narasimhan has received intellectual property interests from a discovery or technology relating to health care.
Paula Williams, Other Mrs. Williams has nothing to disclose.
Stephen Patrick Jung Mr. Jung has received personal compensation for serving as an employee of PTC Therapeutics. Mr. Jung has stock in AbbVie. Mr. Jung has stock in Agilon Health. Mr. Jung has stock in AmeriSourceBergen. Mr. Jung has stock in Abbott Laboratories. Mr. Jung has stock in Aflac. Mr. Jung has stock in Amgen. Mr. Jung has stock in Axa SA. Mr. Jung has stock in BASF. Mr. Jung has stock in Becton, Dickinson and Co. Mr. Jung has stock in BeiGene. Mr. Jung has stock in Boston Scientific. Mr. Jung has stock in Cardinal Health. Mr. Jung has stock in Cigna. Mr. Jung has stock in CNO Financial Group. Mr. Jung has stock in DexCom. Mr. Jung has stock in Icon plc. Mr. Jung has stock in Medtronic plc. Mr. Jung has stock in Elevance Health. Mr. Jung has stock in EXACT Sciences. Mr. Jung has stock in CVS Health. Mr. Jung has stock in Centene. Mr. Jung has stock in Certara. Mr. Jung has stock in Colgate-Palmolive. Mr. Jung has stock in Danaher. Mr. Jung has stock in DuPont de Nemours. Mr. Jung has stock in Edwards Lifesciences. Mr. Jung has stock in Elekta. Mr. Jung has stock in General Electric. Mr. Jung has stock in Genmab A/S. Mr. Jung has stock in Gilead Sciences. Mr. Jung has stock in HCA Healthcare. Mr. Jung has stock in Hologic. Mr. Jung has stock in Illumina. Mr. Jung has stock in Incyte. Mr. Jung has stock in Intuitive Surgical. Mr. Jung has stock in Iqvia Holdings. Mr. Jung has stock in Kimberly Clark. Mr. Jung has stock in Lifestance Health Group. Mr. Jung has stock in McKesson. Mr. Jung has stock in MetLife. Mr. Jung has stock in Moderna. Mr. Jung has stock in Omega Healthcare Investors. Mr. Jung has stock in Omron. Mr. Jung has stock in Proctor & Gamble. Mr. Jung has stock in Reckitt Benckiser Group plc. Mr. Jung has stock in Regeneron Pharmaceuticals. Mr. Jung has stock in Shockwave Medical. Mr. Jung has stock in Siemens AG. Mr. Jung has stock in Siemens Healthineers ADR. Mr. Jung has stock in Straumann Holding ADR. Mr. Jung has stock in Stryker. Mr. Jung has stock in Terumo. Mr. Jung has stock in Thermo Fisher Scientific. Mr. Jung has stock in 3M. Mr. Jung has stock in Unicharm. Mr. Jung has stock in UnitedHealth Group. Mr. Jung has stock in Vertex Pharmaceuticals. Mr. Jung has stock in Walmart. Mr. Jung has stock in Alnylam Pharmaceuticals.
Melissa Wigderson Dr. Wigderson has received personal compensation for serving as an employee of Novartis Gene Therapies. Dr. Wigderson has stock in Novartis.
Mathukumalli Lakshmi Neeharika, MD,MBBS,Other Dr. Neeharika has nothing to disclose.
Karyn Koladicz, MD Dr. Koladicz has received personal compensation for serving as an employee of PTC Therapeutics.
Brooke Marie Marvel Ms. Marvel has nothing to disclose.
Jonathan D. Marra Mr. Marra has received personal compensation for serving as an employee of Novartis Pharmaceuticals Corporation.
Nicolas Marcelo Ballarini, PhD Dr. Ballarini has received personal compensation for serving as an employee of Novartis.
Iulian Alecu, MD Dr. Alecu has stock in Novartis.
Wencheng Li, PhD Dr. LI has received personal compensation for serving as an employee of PTC Therapeutics. Dr. LI has stock in PTC Therapeutics.
Kris Kahlig, PhD Dr. Kahlig has received personal compensation for serving as an employee of Praxis Precisoin Medicines. Dr. Kahlig has stock in Praxis Precision Medicines.
Lyndsey Anderson Anderson, PhD Dr. Anderson has received personal compensation for serving as an employee of Praxis Precision Medicines.
David Ginty, PhD Dr. Ginty has nothing to disclose.
Kiani Jacobs Miss Jacobs has nothing to disclose.
Catherine Susan Franklin, MBBS,Other Dr. Franklin has nothing to disclose.
Anna Mollin Mrs. Mollin has received personal compensation for serving as an employee of PTC Therapeutics. Mrs. Mollin has stock in PTC THERAPEUTICS.
Grace Liu, PhD Dr. Liu has nothing to disclose.
Marla Weetall, PhD Dr. Weetall has received personal compensation for serving as an employee of PTC Therapeutics.
Greg Voronin, Other Dr. Voronin has received personal compensation for serving as an employee of PTC Therapeutics. Dr. Voronin has stock in PTC Therapeutics.
Celine Santiago, PhD Dr. Santiago has nothing to disclose.
William A. Eckert, III, PhD Dr. Eckert has received personal compensation for serving as an employee of Praxis Precision Medicines. Dr. Eckert has received personal compensation for serving as an employee of Janssen Research & Development, LLC. Dr. Eckert has received personal compensation for serving as an employee of Inspire Pharmaceuticals, Inc.. Dr. Eckert has stock in Praxis Precision Medicines. Dr. Eckert has stock in Janssen Research and Development. Dr. Eckert has received intellectual property interests from a discovery or technology relating to health care.
Jennifer Kearney, PhD Prof. Kearney has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Prof. Kearney has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurocrine Biosciences. The institution of Prof. Kearney has received research support from NIH. The institution of Prof. Kearney has received research support from Dravet Syndrome Foundation. The institution of Prof. Kearney has received research support from Lennox Gastaut Foundation. The institution of Prof. Kearney has received research support from Pfizer. The institution of Prof. Kearney has received research support from Neurocrine Biosciences. The institution of Prof. Kearney has received research support from Praxis Precision Medicines. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care. Prof. Kearney has received intellectual property interests from a discovery or technology relating to health care.
Philip Lipari Mr. Lipari has received personal compensation for serving as an employee of PTC Terapeutics.
Jasmine Turna, PhD Dr. Turna has received personal compensation for serving as an employee of PTC Therapeutics. Dr. Turna has stock in PTC Therapeutics.
Brendan Lehnert, PhD Dr. Lehnert has received personal compensation for serving as an employee of Flagship Pioneering.
Agathe Roubertie, PhD,MD Prof. ROUBERTIE has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Prof. ROUBERTIE has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC.
Irina Teplova, PhD Dr. Teplova has nothing to disclose.
Manoj Patel, PhD Dr. Patel has received personal compensation for serving as an employee of GenEP. Dr. Patel has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Gep Ep. Dr. Patel has stock in GenEP.
Robin Alexander, Other Ms. Alexander has nothing to disclose.
Chia Ma, Other Ms. Ma has nothing to disclose.
Gergely Zoltan Papp, MD Dr. Papp has received personal compensation for serving as an employee of F. Hoffmann-La Roche AG.
Shuizhen Zhou, MD, PhD No disclosure on file
Erin Marie Baker, PhD The institution of Dr. Baker has received research support from Praxis Precision Medicines, Inc.
Pravin Wagley Mr. Wagley has nothing to disclose.
Alexis Russell Mrs. Russell has received personal compensation for serving as an employee of PTC Therapeutics. Mrs. Russell has stock in PTC Therapeutics.