Prenatal therapy for spinal muscular atrophy (SMA) with risdiplam has shown promise in a first-of-its-kind study led by Richard Finkel. Roche's Evrysdi has already shown profound benefits in children ...
Roche and PTC Therapeutics' Evrysdi for spinal muscular atrophy (SMA) has been approved by the FDA for younger children with the rare disease, extending its use to include infants less than two months ...
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