Setting goals, adapting, and advocating for himself are all key to columnist Patrick Moeschen's approach to life with ...

DMD treatment SMT-01, a cell replacement therapy, received FDA orphan drug and rare pediatric disease designations.

Rob Stemple is a lifelong advocate for people with disabilities. He was diagnosed with FSHD in 1971 at age 14. Rob struggled with its’ affects for over 50 years. He lost his eyesight in a devastating ...

For a long time, columnist Robin Stemple resisted using a walker to navigate around his home, but eventually, he had to put ...

Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at ...

Last Saturday, I had the honor of participating in the third Singapore Health Patient Advocate Connection event (SPACe) ...

The U.S. Food and Drug Administration granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping DMD ...

Columnist Betty Vertin is going gray — as in, she's decided to stop coloring her hair. Did DMD play a role in this decision, she wonders.

Atamyo has partnered with the Dion Foundation to expand into the U.S. a clinical trial testing the gene therapy ATA-200 for ...

The U.S. Food and Drug Administration has granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping Duchenne muscular dystrophy (DMD) treatment from NS Pharma. Treatments granted ...