2018 Annual Meeting | N2 - Neuroscience in the Clinic: Antisense Oligonucleotide (ASO) Therapy

Date Monday 04/23/18

Time 03:30 PM - 05:30 PM Add To Calendar

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

On Demand This program is not expected to be available in the meeting's On Demand product.

Event Type Neuroscience in the Clinic Session

Director(s) John W. Day, MD, PhD, Richard S. Finkel, MD, FAAN

Topic(s) Neuromuscular and Clinical Neurophysiology (EMG)

Learning Objectives Participants will be able to identify different antisense oligonucleotide chemistries and their relative values. They will understand the biodistribution and pharmacokinetics of some antisense oligonucleotides, and the various ways in which antisense oligonucleotides can impact normal and pathogenic gene expression. They will get an update on recently approved antisense treatments that are now commercially available, as well as those that are currently in development.

CME Available 2.00 CME credits

Core Competencies Patient Care & Procedural Skills, Medical Knowledge

Recommended Audience General Neurologist, Specialist Neurologist, Non-neurologist

Teaching Styles Case-based, Didactic, Panel Discussion

Event Timeline
03:30 PM - 03:35 PM	Luncheon	Introduction John W. Day, MD, PhD
03:30 PM - 05:30 PM	Abstract	001 - Safety and Efficacy of Inotersen in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (NEURO-TTR)
03:30 PM - 05:30 PM	Abstract	002 - Stereopure Antisense Oligonucleotides Preferentially Knockdown G4C2 Repeat-Containing C9ORF72 Transcripts: A Potential Therapeutic Approach for the Treatment of Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) Yuanjing Liu
03:35 PM - 03:50 PM	Speaker	Scientific Content on ASOs Frank Bennett, PhD
03:50 PM - 04:05 PM	Speaker	Case Presentation Richard S. Finkel, MD, FAAN
04:05 PM - 04:20 PM	Speaker	Case Presentation John W. Day, MD, PhD
04:20 PM - 04:30 PM	Speaker	Scientific Wrap-up of Case Presentation Treatments Frank Bennett, PhD
04:30 PM - 04:45 PM	Speaker	N2.001: Safety and Efficacy of Inotersen in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (NEURO-TTR)
04:45 PM - 05:00 PM	Speaker	N2.002: Stereopure Antisense Oligonucleotides Preferentially Knockdown G4C2 Repeat-Containing C9ORF72 Transcripts: A Potential Therapeutic Approach for the Treatment of Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) Robert H. Brown, Jr., MD, DPhil
05:00 PM - 05:30 PM	Q&A	Panel Discussion and Audience Q&A

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.
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.
Frank Bennett, PhD	No disclosure on file
Yuanjing Liu	Yuanjing Liu has received personal compensation for serving as an employee of Wave life sciences . Yuanjing Liu has received stock or an ownership interest from Wave life sciences.
	No disclosure on file
Robert H. Brown, Jr., MD, DPhil	Dr. Brown has received personal compensation for serving as an employee of ApicBio. Dr. Brown has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ApicBio. Dr. Brown 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 Warren Alpert Foundation. Dr. Brown has received stock or an ownership interest from ApicBio. Dr. Brown has received stock or an ownership interest from Amylx. Dr. Brown has received stock or an ownership interest from Imstar. The institution of Dr. Brown has received research support from Angel Fund for ALS Research. The institution of Dr. Brown has received research support from ALS Association. The institution of Dr. Brown has received research support from ALSOne. The institution of Dr. Brown has received research support from NINDS. The institution of Dr. Brown has received research support from ALS Finding A Cure. Dr. Brown has received publishing royalties from a publication relating to health care.
Peter J. Dyck, MD, FAAN	Dr. Dyck has nothing to disclose.
Jean-Cosme Dodart, PhD	Dr. Dodart has received personal compensation for serving as an employee of Vaxxinity. Dr. Dodart has stock in Vaxxinity.
Michael Byrne	No disclosure on file
Annabel K. Wang, MD, FAAN	Dr. Wang has nothing to disclose.
Jason Zhang, PhD	No disclosure on file
Thomas Brannagan III, MD, FAAN	Dr. Brannagan 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. Brannagan 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. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Grifols. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alnylam. The institution of Dr. Brannagan has received research support from Alnylam. The institution of Dr. Brannagan has received research support from Acceleron. The institution of Dr. Brannagan has received research support from Ionis. The institution of Dr. Brannagan has received research support from Pharnext.
Elizabeth Ackermann, PhD	No disclosure on file
Brett Monia, PhD	No disclosure on file
Brenda F. Baker	No disclosure on file
Bradley W. McEvoy	Bradley W. McEvoy has received personal compensation for serving as an employee of Acadia Pharmaceuticals. Bradley W. McEvoy has received stock or an ownership interest from Acadia Pharmaceuticals.
Steven hughes	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.
Merrill Benson	No disclosure on file
Josep Gamez, MD, PhD	The institution of Dr. Gamez has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL Behring. The institution of Dr. Gamez has received research support from Government of Spain (FIS FEDER).
Acary S. Oliveira, MD	No disclosure on file
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.
Fabio Adrian Barroso, MD	Fabio Adrian Barroso, MD has nothing to disclose.
Isabel Conceicao	Isabel Conceicao has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Isabel Conceicao has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sobi.
Chandra Vargeese	No disclosure on file
Violaine Plante-Bordeneuve, MD	Dr. Plante-Bordeneuve has nothing to disclose.
John L. Berk	John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca/IONIS. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eidos/BridgBio. John L. Berk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Intellia Therapeutics. John L. Berk has received research support from Alnylam . John L. Berk has received research support from Ionis. John L. Berk has received research support from Eidos/Bridgbio.
Teresa Coelho, MD	Dr. Coelho has nothing to disclose.
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.