Scholar Rock aims for a 2025 launch of apitegromab in SMA, expecting $2 billion in global revenue and significant growth from upcoming trials and favorable data.

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 ...

Advocates rallied outside the State House on Tuesday for special education funding.The group urged lawmakers to allocate more ...

Crystal Rondeau’s muscle wasting disease has left her with only one finger to press an alarm button or send a text. The ...

who are living with Spinal Muscular Atrophy (SMA) with the support of her mother, sister and teacher. Ms. Keya was felicitated by the Visakha Rasagna Vedika, a cultural organisation, at the Public ...

Haris Beeran urges government intervention to make affordable SMA medicines available in India, addressing the high costs and patent issues.

—A new study analyzed health-related quality of life (HRQoL) using 5 generic and neuromuscular disease-specific rating scales in German children with spinal muscular atrophy (SMA), most of whom ...

Typically, patients do not attend these meetings, with their family or caregivers representing them, says Unnikrishnan. But, in the case of Maldivian disability activist Mohamed Raishan Ahmed's ...

Positive results from its Phase III STEER study mean Novartis could boost sales of its OAV101 IT drug for spinal muscular ...

Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene ...

Upper House member Haris Beeran has said the current costs of medicines approved for SMA are 'beyond the reach of the central ...