The decision marks a significant milestone after both medicines were previously made available through managed access agreements while additional evidence on their effectiveness was gathered. SMA is a ...

Spinal muscular atrophy is a rare, genetic condition that causes progressive muscle weakness and loss of movement, and can ...

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 ...

The family of a three-year-old girl with a rare muscle disease are facing a bill of £7,000 for adaptations to their home.

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...

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 ...

Medicines watchdog approves two treatments for patients with spinal muscular atrophy ...

Jesy Nelson shares heartfelt tribute to her ‘fighter’ twins on first birthday - The singer’s twins Ocean Jade and Story ...

If approved, Itvisma (intrathecal onasemnogene?abeparvovec) will be first and only gene replacement therapy for children two years and older, teens and adults with SMA in the European UnionItvisma dem ...

Pop star Jesy Nelson has backed calls for routine screening, as Oxford University launches a study to help infants with ...