A 5-year-old boy who was born with a rare genetic condition is now able to walk by himself, his mother has said, after receiving the world's most expensive drug, which costs around $2.4 million.

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 lead to severe physical disability, paralysis and life ...

Spinal muscular atrophy explained after Jesy Nelson reveals her twins’ diagnosis - The singer shared an emotional update about her twins’ health on Sunday and that ‘time is of the essence with this di

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA). The study's results add further support for gene ...

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no signs at all two and a half years after birth.

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with ...

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended first-line nusinersen, new data showed. Those treated with ...