British singer Jesy Nelson recently took to Instagram to share a devastating update about her 8-month-old twin daughters.

Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease. This is a ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both ...

Health Secretary was responding to news shared by the former Little Mix star that her twin daughters have been diagnosed with ...

Please provide your email address to receive an email when new articles are posted on . The study examined 26 newborns with spinal muscular atrophy given Evrysdi. Data showed 81% of babies were able ...

A Paediatric neurologist shares insight into the condition after former Little Mix singer Jesy Nelson revealed her twins have ...

NBS programs are fairly new in the U.S., so not much is known about variations among them or about provider practice patterns for newborns with SMA. In a survey of providers and state NBS programs, ...

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

DUBLIN--(BUSINESS WIRE)--The "Spinal Muscular Atrophy (SMA) Treatment Market Size, Share & Trends Analysis Report By Route of Administration (Oral, Intrathecal), By Treatment (Gene Therapy, Drug), By ...

The BBC's Ben Morris describes living with spinal musclar atrophy, after Little Mix's Jessy Nelson spoke about it.