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Session 1C: Clinical Track: Neuromuscular
Session Chair(s)
Akshay K. Vaishnaw, MD, PhD
Chief Medical Officer
Alnylam Pharmaceuticals, Inc., United States
The objectives of this session are to highlight recent progress utilizing RNAi and ASO approaches to address high unmet need disorders of the nervous system. The clinical focus will be on two programs in Phase 3, comprising ALN-TTR, a systemically delivered RNAi therapeutic for TTR amyloidosis, a disorder of peripheral nerves, and ISIS-SMNRx, an intrathecally administered ASO for spinal muscular atrophy, a pediatric motor neuron disorder. A third presentation will focus on another neurodegenerative disorder, Huntington’s disease, examining recent preclinical progress where again an intrathecally administered ASO is being developed.
Speaker(s)
Antisense Oligonucleotides Drugs For the Treatment of Neurodegenerative Diseases
Laurence Mignon, PhD
Ionis Pharmaceuticals, Inc., United States
Director, Clinical Development
Antisense Oligonucleotide Therapies for the Treatment of Huntington’s Disease
Holly Kordasiewicz
Isis Pharmaceuticals, United States
Update on ALN-TTR Programs for the Treatment of Transthyretin Amyloidosis
Jared A. Gollob, MD
Alnylam Pharmaceuticals, Inc., United States
Vice President, Clinical Research
Panel Discussion
All Session Speakers, United States
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