The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NS-051/NCNP-04, an exon-skipping therapy ...

Columnist Patrick Moeschen shares what he wishes doctors understood about treating patients with rare diseases such as ...

One thing I love about living in the Midwest is enjoying all four seasons. The last week of summer is nearly over, and autumn officially begins next week. On a walk yesterday, I noticed the first ...

Patients aged 10-50 are being sought for a clinical trial testing SAR446268, a gene therapy for myotonic dystrophy type 1 ...

Living with Duchenne muscular dystrophy means every trip to the hospital is key to my survival. From heart scans to routine medical appointments, leaving home is never simple. I rely on my mom to ...

I was diagnosed with Duchenne muscular dystrophy at age 3. By the time I was 10, I was exclusively using a power wheelchair for mobility — and I’ve been rolling ever since. Living with a chronic ...

A combined regimen of testosterone and growth hormone improves muscle mass, strength, and mobility in men with FSHD, a study ...

Mallory Dupree hasn't let a Duchenne muscular dystrophy diagnosis keep her from living the life she wants to live.

Mallory Dupree lives with Duchenne muscular dystrophy and whose experience in Duchenne advocacy began as a teen, meeting with members of Congress to advocate for the passage of the MD-CARE Act. Since ...

Duvyzat's approval stemmed from the Phase 3 EPIDYS clinical trial that tested the therapy against a placebo in 179 boys with ...

Finding humor and joy in life might not change your situation, but it can change your perspective on it, columnist Robin ...

To live with dignity, columnist Shalom Lin argues, young adults with disabilities in Singapore deserve stronger housing ...