Healio2h
Gene therapy improves motor function in spinal muscular atrophy at 52 weeks
Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...
Rheumatology Advisor7h
Apitegromab Gets Priority Review for Spinal Muscular Atrophy
The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.
Film on boy's rare disability nominated for Bafta
A film about a Kent boy who lives with a rare condition has been nominated for a Bafta TV award. Sky's Disability & Me ...