Spinal Muscular Atrophy: Why a New Antisense Therapy is Moving to Phase 3 Trials
John Bodensteiner (Consultant, Child and Adolescent Neurology), our Paediatric Neurology Section Editor for US Neurology, discusses the impact recent advances in molecular and genetic diagnosis have had in paediatric neurology on conditions such as Duchenne muscular dystrophy and spinal muscular atrophy. Speaker disclosure: John Bodensteiner has nothing to disclose in relation to this video interview. […]
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)? […]
Prof. John Bodensteiner, from the US Neurology Editorial Board, discusses the diagnostic benefits of nerve biopsies in pediatric neurology and reviews the most exciting developments in spinal muscular atrophy and Duchenne muscular dystrophy. Speaker disclosures: Data Safety Review Board Biogen. Filmed at the American Academy of Neurology (AAN) Annual Meeting, Boston, US, […]
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