Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Nusinersen provides continued benefits to presymptomatic children with SMA

Publish date:: October 22, 2020

Key clinical point: Nusinersen provides long-term beneficial changes to the disease course in children with spinal muscular atrophy who initiated treatment when they were presymptomatic.

Major finding: About 84% of study participants have achieved a maximum score on the CHOP INTEND scale.

Study details: An interim analysis of data for 25 infants with spinal muscular atrophy enrolled in the ongoing open-label NURTURE study.

Disclosures: Biogen is sponsoring the study.

Citation:

Chin R et al. CNS-ICNA 2020, Abstract PL78.

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