2023 Annual Meeting | S14 - General Neurology: Emerging Therapies

Date Monday 04/24/23

Time 01:00 PM - 03:00 PM EST Add To Calendar

Session Format This program will be presented both in-person and online

On Demand This program will be available in the meeting's On Demand product.

Event Type Scientific Platform Session

Moderator(s) Marie Y. Davis, MD, PhD, Sarah Wahlster, MD

Topic(s) General Neurology

Learning Objectives 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.

CME Available 2.00 CME credits

Recommended Audience Fellow, Resident, General Neurologist, Specialist Neurologist, Non-neurologist, Advanced Practice Provider, Neurohospitalist, Medical Student

Teaching Styles Didactic

Event Timeline
01:00 PM - 01:12 PM EST	Abstract	001 - NfL Levels Significantly Decrease in Response to Treatment with Patisiran or Vutrisiran in hATTR Amyloidosis with Polyneuropathy Michael J. Polydefkis, MD, FAAN
01:12 PM - 01:24 PM EST	Abstract	002 - Comparison of Therapeutic Efficacy of Gene Therapy for Tuberous Sclerosis Type 2 with Standard of Care Everolimus in Preclinical Model Edwina Abou Haidar, MBBS, MS
01:24 PM - 01:36 PM EST	Abstract	003 - Comparison of Efficacy Outcomes with Vutrisiran vs. Patisiran in hATTR Amyloidosis with Polyneuropathy: Post-hoc Analysis of the HELIOS-A Study Michael J. Polydefkis, MD, FAAN
01:36 PM - 01:48 PM EST	Abstract	004 - A Phase 1, First-in-human, Randomized, Double-blind, Placebo-controlled, Single and Multiple Ascending Intravenous Dose Study of a novel TREM2 agonist (VGL101) in Healthy Volunteers (HVs) Andreas Meier, MD
01:48 PM - 02:00 PM EST	Abstract	005 - Effect of Patisiran on Polyneuropathy and Cardiomyopathy in Patients with hATTR Amyloidosis with V122I/T60A Variants: A Phase 4 Observational Study
02:00 PM - 02:12 PM EST	Abstract	006 - Enabling Subcutaneous Dosing of Gantenerumab in Alzheimer’s Disease Janice W. Smith, PhD
02:12 PM - 02:24 PM EST	Abstract	007 - Longer Milestone-Free Time in Patients With Amyotrophic Lateral Sclerosis Treated With IV Edaravone vs Not Treated With IV Edaravone: Results From a US Administrative Claims Analysis Malgorzata Ciepielewska
02:24 PM - 02:36 PM EST	Abstract	008 - Therapeutic Potential of Fosgonimeton, a Small-Molecule Positive Modulator of the Neurotrophic HGF/MET Pathway, in Neurodegenerative Conditions Kevin Church, PhD
02:36 PM - 02:48 PM EST	Abstract	009 - Angubindin-1 opens the blood–brain barrier in vivo for delivery of antisense oligonucleotide to the central nervous system Hiroya Kuwahara, MD, PhD
02:48 PM - 03:00 PM EST	Abstract	010 - Remote multimodal monitoring of motor neuron disease progression using wearable sensors and digital assessments Ashkan Vaziri, PhD

Faculty Disclosures
Jeffrey Zhang	No disclosure on file
Emre Aldinc	Emre Aldinc has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Emre Aldinc has stock in Alnylam Pharmaceuticals.
Davide Pareyson, MD	No disclosure on file
	No disclosure on file
Kevin Church, PhD	Dr. Church has received personal compensation for serving as an employee of Athira Pharma. Dr. Church has stock in Athira Pharma. Dr. Church has received intellectual property interests from a discovery or technology relating to health care.
Jewel Johnston	Dr. Johnston has received personal compensation for serving as an employee of Athira Pharma, Inc. Dr. Johnston has stock in Athira Pharma, Inc. An immediate family member of Dr. Johnston has stock in Athira Pharma, Inc. Dr. Johnston has received intellectual property interests from a discovery or technology relating to health care.
Xandra O. Breakefield, PhD	Dr. Breakefield has nothing to disclose.
Ram Kinker Mishra	No disclosure on file
Malgorzata Ciepielewska	Malgorzata Ciepielewska has received personal compensation for serving as an employee of Mitsubishi Tanable Pharma America, Inc..
Francy Y. Shu, MD	Dr. Shu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH. Dr. Shu has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alnylam. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for The CM Group. Dr. Shu has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for T&Associates. The institution of Dr. Shu has received research support from Biogen. The institution of Dr. Shu has received research support from Alnylam.
Zachary Simmons, MD, FAAN	Dr. Simmons has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cytokinetics. The institution of Dr. Simmons has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amylyx. Dr. Simmons has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Simmons has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clene. Dr. Simmons 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. Simmons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Corcept. The institution of Dr. Simmons has received personal compensation in the range of $50,000-$99,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Wiley. The institution of Dr. Simmons has received research support from MGH. The institution of Dr. Simmons has received research support from MT Pharma. The institution of Dr. Simmons has received research support from University of Kansas. The institution of Dr. Simmons has received research support from ALS Association. The institution of Dr. Simmons has received research support from Clene.
Chafic Y. Karam, MD	Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Karam has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neuroderm.
Andree-Anne Berthiaume	No disclosure on file
David Stiles, PhD	Dr. Stiles has received personal compensation for serving as an employee of Vigil Neuroscience. Dr. Stiles has received personal compensation for serving as an employee of Vertex Pharmaceuticals.
Michael J. Polydefkis, MD, FAAN	Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharmaceutical . Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen-Idec. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer.
Sharay Setti	No disclosure on file
David D. Adams	David D. Adams has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ALNYLAM. David D. Adams has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer.
Hiroya Kuwahara, MD, PhD	Dr. Kuwahara has nothing to disclose.
Ying Liu	No disclosure on file
Frank Birklein, MD	Dr. Birklein has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. The institution of Dr. Birklein has received research support from Alnylam.
Ashkan Vaziri, PhD	Dr. Vaziri has received personal compensation for serving as an employee of Biosensics.
John Vest	John Vest has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. John Vest has received stock or an ownership interest from Alnylam Pharmaceuticals. John Vest has received intellectual property interests from a discovery or technology relating to health care.
Evan Thackaberry	No disclosure on file
Jose Joaquin Casado	No disclosure on file
Kelley E. Capocelli, MD	Dr. Capocelli has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Dr. Capocelli has stock in Alnylam Pharmaceuticals.
Edwina Abou Haidar, MBBS, MS	Dr. Abou Haidar has nothing to disclose.
Robert Taylor	No disclosure on file
Andrew Geronimo	No disclosure on file
Yessar M. Hussain, MD	The institution of Dr. Hussain has received research support from Alnylam Pharma.
Tetsuya Nagata, MD, PhD	Dr. Nagata has nothing to disclose.
Senda Ajroud-Driss, MD, FAAN	Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx Pharmaceuticals. Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Ajroud-Driss has received research support from ALSA. Dr. Ajroud-Driss has received publishing royalties from a publication relating to health care.
Urvi G. Desai, MD, FAAN	Dr. Desai has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Desai 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. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Desai has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx.
Madeline Merkel	Madeline Merkel has received personal compensation for serving as an employee of Alnylam Pharmaceuticals.
Sherif Reda	No disclosure on file
Yoshiki Sekijima, MD, PhD	Yoshiki Sekijima, "MD, PhD" has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alnylam. Yoshiki Sekijima, "MD, PhD" has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Pfizer.
Marcia Waddington Cruz, MD	Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sobi. Dr. Waddington Cruz 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. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ionis. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NovoNordisk.
Spyridon Papapetropoulos, MD, PhD	Dr. Papapetropoulos has received personal compensation for serving as an employee of Vigil Neuroscience . Dr. Papapetropoulos has received personal compensation for serving as an employee of Bionomics Ltd. Dr. Papapetropoulos 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 Lipocine Inc. Dr. Papapetropoulos has stock in Vigil Neuroscience. Dr. Papapetropoulos has stock in Lipocine. Dr. Papapetropoulos has stock in Bionomics Ltd.
Rajasimhan Rajagovindan, PhD	Dr. Rajagovindan has received personal compensation for serving as an employee of Biogen. Dr. Rajagovindan has received personal compensation for serving as an employee of Vigil. Dr. Rajagovindan has stock in Biogen. Dr. Rajagovindan has stock in Vigil .
Takanori Yokota, MD, PhD	Dr. Yokota has nothing to disclose.
Alexandra Geffrey, MD	Dr. Geffrey has received personal compensation for serving as an employee of Massachusetts General Hospital.
Marie Y. Davis, MD, PhD	Dr. Davis has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Davis has received research support from NIH NINDS. The institution of Dr. Davis has received research support from University of Washington Institute for Stem Cell and Regenerative Medicine. The institution of Dr. Davis has received research support from VA BLRD. Dr. Davis has received personal compensation in the range of $500-$4,999 for serving as a study section grant reviewer with NIH. Dr. Davis has received personal compensation in the range of $500-$4,999 for serving as a Grant reviewer with Parkinson's Foundation.
Sarah Wahlster, MD	Dr. Wahlster has nothing to disclose.
Ali Toumadj, PharmD	No disclosure on file
Janice W. Smith, PhD	Dr. Smith has received personal compensation for serving as an employee of Roche Products Ltd. Dr. Smith has stock in Hoffman La Roche.
Andreas Meier, MD	Dr. Meier has received personal compensation for serving as an employee of Vigil Neuroscience. Dr. Meier has received personal compensation for serving as an employee of Voyager Therapeutics. Dr. Meier has stock in Vigil Neuroscience. Dr. Meier has stock in Voyager Therapeutics.