Ellie Kerrigan, who has spinal muscular atrophy, shared her journey through Alabama sorority recruitment on TikTok.

If approved, Itvisma (intrathecal onasemnogene abeparvovec) will be first and only gene replacement therapy for children two ...

A NEW screening for spinal muscular atrophy (SMA) is now being offered, included in the heel prick test, to all newborns in ...

Cenrifki prevents MS disease progression and Itvisma delivers a healthy gene-to-cell nuclei, addressing the root cause of SMA.

(SMA) causes progressive muscle weakness and, without treatment, can limit life expectancy to just two years. The milestone means the petition, to include SMA in post-birth baby checks for serious ...

According to the Cleveland Clinic, spinal muscular atrophy (SMA) is a group of inherited neuromuscular disorders ...

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Many new gene therapy treatments aren’t as profitable as predicted. But the reasons why don’t quite fit Goldman Sachs’ ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

Spinal muscular atrophy (SMA) is a rare, genetic condition that, across all types, affects ~1,600 people in the UK.1 It can cause muscle weakness which gets worse over time, causing movement, ...