Type 3 and 4 are typically for the older age groups. And type 4 is mainly in the adult onset. They do have the ability to walk. However, over time, they may lose their ability to walk and will ...

More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...

Health activists demand government action to reduce the cost of SMA medicines, citing constitutional obligations and ...

Back to Healio Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease ...

Scholar Rock is asking the U.S. Food and Drug Administration to approve apitegromab for people with spinal muscular atrophy.

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

The health activists argue that the government is empowered with effective law and policy tools and therefore should ...

Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for ...