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

Princess Kate played with seriously ill children and chatted with bereaved families on a surprise visit to Wales on Thursday.

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

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.

Scholar Rock , a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein ...

Biogen has announced that the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) have started ...

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 ...

MNDs are neurological disorders that destroy motor neurons, the cells that control skeletal muscle activity such as walking, breathing, speaking, and swallowing.