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

Scholar Rock (SRRK), a late-stage biopharmaceutical company developing innovative treatments for spinal muscular atrophy, ...

Learn more about whether Ionis Pharmaceuticals, Inc. or PTC Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

Problems with chemical messengers like dopamine were tied to posture issues in SMA in a mouse study, but levodopa therapy may ease symptoms.

One-year-old Asmika Das from Ranaghat, Nadia, is suffering from a fatal disease called Spinal Muscular atrophy.

Filmmaker Reid Davenport weaves a powerful first-person perspective with a journalistic investigation about disability ...

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

The partners will adapt Eukarÿs' C3P3 engineered enzyme tech to work with adeno-associated virus vectors to boost biomanufacturing yields.

An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...

In a good-news-bad-news week for Biogen, the company will cut an undisclosed number of employees, just as a higher dose of ...