Health news
The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for risdiplam for use in presymptomatic infants less than 2 months of age with spinal muscular atrophy (SMA).…
Lessons learned from various presymptomatic neurodegenerative diseases may provide opportunities for early intervention, and perhaps even disease prevention of amyotrophic lateral sclerosis (ALS), according to a review published in the journal Brain. Studies have shown…
The Food and Drug Administration (FDA) has approved several revisions to the prescribing information for Zolgensma (onasemnogene abeparvovec-xioi). Zolgensma is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than…
Among infants with type 1 spinal muscular atrophy (SMA), treatment with risdiplam was associated with improvements in motor function and meeting developmental motor milestones, according to study results published in The New England Journal of…
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