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 ...

As a parent and caregiver to three sons with DMD, columnist Betty Vertin's definition of what success looks like has changed over the years.

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.

Governor Maura Healey’s budget proposal includes major cuts to health and social service programs, including two residential ...

Learn about the world’s most expensive watches, from Jackie Kennedy’s Cartier to Paul Newman’s Rolex Daytona. Which one tops $31M?

One of the most anticipated and noteworthy wine events of the year, Napa in Newport, returns to the Pendry in Fashion Island on March 1, and it promises to be another stellar evening of superb wines ...

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 ...