Satellos Bioscience is planning a Phase 2 trial in the U.S. to test SAT-3247 in children with Duchenne who are able to walk.

Adaptive clothing for Duchenne muscular dystrophy offers comfort, ease, and independence for those with muscle weakness and ...

The FDA has granted orphan drug designation to CRD-003, an investigational gene therapy for limb-girdle muscular dystrophy ...

Columnist Robin Stemple didn't pay much attention to a random bug bite at first, but his situation dramatically worsened.

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 (DM1).

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 ...

Last Sunday, my girlfriend, Amanda, and I co-led an art workshop that brought us both joy. The accessible art journaling session, “Why your story matters: Where art meets wellness,” was held at the ...