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2016 Annual Meeting | General Neurology: Therapeutics

Wednesday 04/20/16
06:30 AM - 08:30 AM Add To Calendar
This program will be presented both in-person and online
This program is not expected to be available in the meeting's On Demand product.
Scientific Platform Session
General Neurology
2.00 CME credits
Event Timeline
06:30 AM - 08:30 AM Abstract 001 - Edaravone, a Free Radical Scavenger, Delays Symptomatic and Pathological Progression of Wobbler Mouse Motor Neuron Disease
Ken Ikeda, MD, PhD
06:30 AM - 08:30 AM Abstract 002 - Hematopoietic Stem Cell Transplantation in Late-Onset Krabbe Disease Halts Demyelination and Axonal Loss: A 4-Year Longitudinal Case Study
Cornelia Laule, PhD
06:30 AM - 08:30 AM Abstract 003 - Phase 2 Open-Label Extension Study (OLE) of Patisiran, an Investigational RNAi Therapeutic for Familial Amyloid Polyneuropathy (FAP)
David D. Adams
06:30 AM - 08:30 AM Abstract 004 - Functional and Imaging Studies of the Selective Inhibitor of Nuclear Export (SINE) Compound KPT-350 in a Clinically Relevant Murine Model of Closed Head Injury (CHI)
06:30 AM - 08:30 AM Abstract 005 - Phase 1 Safety and Pharmacokinetics of the Novel Enzyme Replacement Therapy neoGAA in Treatment-Naïve and Alglucosidase Alfa-Treated Late-Onset Pompe Disease Patients
Jaya R. Trivedi, MD, FAAN
06:30 AM - 08:30 AM Abstract 006 - Phase 1 Exploratory Efficacy of the Novel Enzyme Replacement Therapy NeoGAA in Treatment-Naïve and Alglucosidase Alfa-Treated Late-Onset Pompe Disease Patients
Loren D. Pena
06:30 AM - 08:30 AM Abstract 007 - The Effect of Bolus Volume and Mechanical Forces on the Biodistribution of ASOs Following Lumbar Intrathecal Administration in Cynomolgus Monkeys
Daniel A. Wolf, PhD
06:30 AM - 08:30 AM Abstract 008 - Kinetics of ASO Distribution and Pharmacodynamics in the CNS after an Intrathecal Bolus Dose in Rat
Eric Swayze, PhD
Faculty Disclosures
Vishunmurthy S. Hedna, MD No disclosure on file
Swetha Renati, MD Dr. Renati has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ischemia View. Dr. Renati has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bayer. Dr. Renati has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Wettermark Keith, LLC. Dr. Renati has received personal compensation in the range of $500-$4,999 for serving as a Moderator with PRIME 好色先生.
Jean Pouget, MD, PhD No disclosure on file
Yasuo Iwasaki, MD No disclosure on file
Richard J. Barohn, MD, FAAN Dr. Barohn has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for NuFactor. The institution of Dr. Barohn has received research support from FDA OPD R01. Dr. Barohn has received intellectual property interests from a discovery or technology relating to health care.
Alan Pestronk, MD, FAAN The institution of Dr. Pestronk has received research support from Jain. The institution of Dr. Pestronk has received research support from Fulcrum. The institution of Dr. Pestronk has received research support from Argenyx. The institution of Dr. Pestronk has received research support from NeuroNext.
John Vissing, MD Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne Therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Solid Biosciences. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AskBio. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity Therapeutics. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Italfarmaco. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Prof. Vissing 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. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Biopharma. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion Pharmaceuticals. Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson & Johnson (Janssen). Prof. Vissing has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Edgewise Therapeutics. The institution of Prof. Vissing has received research support from Rigshospitalet. The institution of Prof. Vissing has received research support from UCB Pharma. The institution of Prof. Vissing has received research support from Sanofi. The institution of Prof. Vissing has received research support from Roche. The institution of Prof. Vissing has received research support from AnJii.
Claude J. Desnuelle, MD No disclosure on file
Pascal Laforet No disclosure on file
Shafeeq Ladha, MD Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Ladha has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Ladha has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech. Dr. Ladha has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Ladha has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurosense. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rapa Therapeutics. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen. Dr. Ladha has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Sanofi. Dr. Ladha has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Genentech. The institution of Dr. Ladha has received research support from Biogen. The institution of Dr. Ladha has received research support from Sanofi.
Daniel T. Laskowitz, MD 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.
Ajay Verma, MD, PhD No disclosure on file
Kirsten L. Gruis, MD No disclosure on file
No disclosure on file
Jaya R. Trivedi, MD, FAAN The institution of Dr. Trivedi has received research support from Avidity.
Barry Byrne Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rocket Pharma. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving as an officer or member of the Board of Directors for Muscular Dystrophy Association.
Ken Ikeda, MD, PhD No disclosure on file
Ole Suhr The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam . The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Prothena. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Intellia. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akces. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alnylam. Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Akcea. The institution of Ole Suhr has received research support from Swedish Heart and Lung Foundation.
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.
Ans Van Der Ploeg The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi Genzyme . The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics . The institution of Ans Van Der Ploeg has received research support from Sanofi Genzyme.
Philip Van Damme The institution of Dr. Van Damme has received research support from CSL Behring.
Cornelia Laule, PhD The institution of Dr. Laule has received research support from Multiple Sclerosis Society of Canada. The institution of Dr. Laule has received research support from Natural Sciences and Engineering Research Council of Canada (NSERC). The institution of Dr. Laule has received research support from International Collaboration on Repair Discoveries (ICORD).
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.
David K. Li, MD Dr. Li has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Li has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Biogen. Dr. Li has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Academy of Health Care Learning. Dr. Li has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi-Genzyme. Dr. Li has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Consortium of MS Centers.
Jared Gollob, MD No disclosure on file
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.
Akshay Vaishnaw Akshay Vaishnaw, 22867 has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Akshay Vaishnaw, 22867 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 Editas Medicine. Akshay Vaishnaw, 22867 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 Scholar Rock. Akshay Vaishnaw, 22867 has received stock or an ownership interest from Alnylam Pharmaceuticals.
Rick Falzone No disclosure on file
Daniel A. Wolf, PhD No disclosure on file
Benedikt Schoser Benedikt Schoser has received personal compensation for serving as an employee of Alexion. Benedikt Schoser has received personal compensation for serving as an employee of Argenx. Benedikt Schoser has received personal compensation for serving as an employee of Amicus. Benedikt Schoser has received personal compensation for serving as an employee of Astellas. Benedikt Schoser has received personal compensation for serving as an employee of Denali. Benedikt Schoser has received personal compensation for serving as an employee of Avidity . Benedikt Schoser has received personal compensation for serving as an employee of Sanofi. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Taysha. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astellas. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Encoded. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Kedrion.
Loren D. Pena No disclosure on file
Irene Vavasour, PhD Dr. Vavasour has nothing to disclose.
Eric Swayze, PhD No disclosure on file
Eugen Mengel No disclosure on file
Sharon Tamir No disclosure on file