American Academy of Neurology

Big Impact in Bio: Funding the Translation From Bench to Bedside at the American Academy

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At a meeting of the American Academy of Neurology last year, Megan Krench saw something remarkable: Children, previously unable to move their head or sit up, were turning their heads to look at their parents, sitting up, and even walking. It was a video of a company’s clinical trial for a gene therapy to treat a devastating childhood neuromuscular disorder, spinal muscular atrophy. Infants with this disease typically never reach any developmental milestones, and most die by age two.

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