The mother of a four-year-old boy with a rare muscle disease says screening newborn babies for the condition could "save ...

Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology ...

Edgewise Therapeutics, Inc. (NASDAQ:EWTX), a biotechnology company focused on developing treatments for rare muscle disorders, recently unveiled promising results from its sevasemten program for ...

Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology and functional decline in a mouse model of facioscapulohumeral muscular ...

Novartis (NYSE:NVS) (OTCPK:NVSEF) (TSX:NVS:CA) said Sunday it will acquire California-based Avidity Biosciences (NASDAQ:RNA) in a $12 billion all-cash transaction, strengthening the Swiss ...

A rare condition called Eosinophilic Fasciitis caused a 42-year-old engineer severe pain and stiffness. Doctors identified ...

Swiss drugmaker Novartis on Sunday said it agreed to acquire U.S. biotech firm Avidity Biosciences for about $12 billion in cash, as the company looks to bolster its portfolio of treatments for rare ...

2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for ...

ST. PAUL, Minn., May 25 (UPI) --A new rapid blood test for newborns could potentially detect genetic mutations linked to thousands of rare diseases all at once, greatly improving on current ...

As someone living with a rare disease in Ohio, I know how fragile access to life-saving treatment can be. A bill to help patients with rare or life-threatening illnesses is currently being debated in ...

- All Evaluable Patients in the 3.4 mg/kg Cohort Treated with DYNE-101 Q4W Demonstrated Consistent Splicing Correction with a 19% Mean Improvement Across 22-Gene Panel at 3 Months - - Improvement in ...