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

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

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

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

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

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

While I was driving, walking, and living independently in high school, he’d had the opposite experience. For Andrew, his high school days consisted of bone breaks, personal care assistants, hospital ...

Patients aged 10-50 are being sought for a clinical trial testing SAR446268, a gene therapy for myotonic dystrophy type 1 (DM1).

My name is Patrick, and I’m a 31-year-old man living with Duchenne muscular dystrophy. I live with my parents because I require 24-hour care. I need help with almost everything in my day-to-day life, ...

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 ...