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.

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 Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand into the U.S. a clinical trial of ATA-200, a potential gene therapy for limb-girdle muscular ...

SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). The cell replacement therapy ...

In my recent columns, I’ve shared several challenges we’ve faced in the past months of my family’s journey with Duchenne muscular dystrophy (DMD). My accessible van will be in the shop for the ...