Richard Finkel (Nemours Children’s Hospital), recipient of this year’s Sidney Carter Award in Child Neurology, speaks to us on the topic of his presentation during the AAN 2018 Presidential Plenary Session, entitled: Spinal Muscular Atrophy Is a Treatable Neurodegenerative Disease.
Questions
1. What is the mechanism of action of nusinersen in spinal muscular atrophy (SMA)? (0:11)
2. What were the major findings of the ENDEAR study? (0:48)
3. Could you tell us a little about the use of gene replacement therapy in SMA? (1:21)
4. What are the relative benefits and limitations of the two new approaches? (2:14)
5. Can you summarize some of the other therapies currently under investigation for SMA? (3:20)
6. What are the implications for SMA in the future? (4:33)
Speaker disclosures: Richard Finkel has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with consulting fees and travel costs from AveXis, Biogen, Catabasis, Ionis, Mitobridge, and Summit. Dr. Finkel’s institution has received research support to perform clinical trials from Biogen, BMS, Catabasis, Cytokinetics, Ionis, Lilly, ReveraGen, Sarepta, and Summit.
Filmed at the American Academy of Neurology (AAN) Annual Meeting 2018, Los Angeles, CA, US, April 2018.
