Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.

The West Texas Rehab Telethon will be taking place this weekend. This event is to give back to people and facilities that care for people in need.

Learn about the world’s most expensive watches, from Jackie Kennedy’s Cartier to Paul Newman’s Rolex Daytona. Which one tops ...

A new study published in the peer-reviewed journal Muscle & Nerve shows that full-spectrum cannabidiol (CBD) oil may help ...

The Perrysburg gymnastics team hosted the area’s only All Around Invitational on January 20 — the Noah Ninja’s meet: ...

Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

Initiation of Phase 1b study using SGT-212 for the treatment of patients with Friedreich's Ataxia expected in 2H25. Click ...

Company Anticipates Submitting for U.S. Accelerated Approval in H1 2026 -WALTHAM, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, ...

Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX) recently disclosed in an 8-K filing with the Securities and Exchange Commission on January 13, 2025, that the company has posted a corporate presentation ...

As the year gets underway, analysts and biotech executives highlight cell therapy’s pivot from oncology to autoimmune ...