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.

CAMBRIDGE, Mass. - Scholar Rock (NASDAQ: SRRK), a biopharmaceutical company with a market capitalization of $3.96 billion and an impressive 362.73% stock price surge over the past six months, ...

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 ...

The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...

KOCHI: Residents of Cherai are rallying to support two-and-a-half-year-old Adharv, who is suffering from Spinal Muscular ...

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.