|Event Time:||Sunday April 22, 2018 1:00 pm to 3:00 pm|
|Description:||A group of abstracts covering a similar topic presented in an oral format. Presentations are eight minutes in length with four minutes of question and answer. Each session concludes with 25 minutes of a discussant bringing additional context to one or more of the abstracts.|
|2:35 PM - 3:00 PM||Discussion||Faculty|
|1:00 PM||001||Characterization of Atxn2 in ALS Patient Samples and Insight into ASO Therapy||
Dr. Taylor has nothing to disclose.
|1:12 PM||002||Quantitative Determination of SMN2 Copy Number using Next Generation Sequencing and Correlation to Disease Severity||
Dr. Westbrook has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Invitae.
|1:24 PM||003||Effects of Elamipretide in Adults with Primary Mitochondrial Myopathy: a Phase 2 Double-Blind, Randomized, Placebo-Controlled Crossover Trial (MMPOWER-2)||
Bruce Cohen, MD, FAAN
Dr. Cohen has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Stealth Biotheraputics, Modis (aka Meves) Pharmaceuticals, AAN for CPT Consulting and Educational Activities. Dr. Cohen has received research support from Stealth Biotherapeutics, BioElectron Technologies, Reata Pharmaceuticals, Horizon Pharmaceuticals.
|1:36 PM||004||Modulation of Transcriptomic Changes in Alzheimer Disease by Ethnicity||
Sathesh K. Sivasankaran
Dr. Sivasankaran has nothing to disclose.
|1:48 PM||005||Canagliflozin Associated with MELAS Pseudoexacerbation||
Dr. Dewan has nothing to disclose.
|2:00 PM||006||Inotersen Improves Norfolk Quality of Life-Diabetic Neuropathy Measures in Patients With Hereditary Transthyretin Amyloidosis with Polyneuropathy in the Phase 3 Study NEURO-TTR||
Michael J. Polydefkis, MD, FAAN
Dr. Polydefkis has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Ionis, Alnylam, Vertex, Chromocell. Dr. Polydefkis has received compensation for serving on the Board of Directors of Travel-Ionis and Pfizer. Dr. Polydefkis has received research support from Pfizer, Ionis, and Alnylam .
|2:12 PM||007||Variation of Penetrance estimates in a wide spectrum of TTR-FAP families: implication for management of carriers||
Dr. Gorram has nothing to disclose.
|2:24 PM||008||Glycemic Control Boosts Glucosylated Nanocarrier Crossing the BBB into the Brain||
Hiroya Kuwahara, MD, PhD
Dr. Kuwahara has nothing to disclose.