Spinal Muscular Atrophy: Four trials to watch over the next 12 months

In 2016, the first treatment for spinal muscular atrophy (SMA) was approved. Seven years on, there are just five marketed drugs for the indication according to GlobalData’s Pharmaceutical Intelligence Centre. More treatments are effective in type 1 SMA but those diagnosed with type 2 or 3 continue to struggle to access treatment. Those with Kennedy’s disease, known as Spinal and Bulbar Muscular Atrophy (SBMA), have no effective treatments that have been approved.