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 ...

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.

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 ...

Betty Vertin is a mother and writer living in rural Hastings, Nebraska, with her husband and seven children. Betty is a caregiver to three sons, Max, Rowen, and Charlie. Her oldest was diagnosed with ...

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

Columnist Shalom Lim reflects on how he and his late brother, Isaac, left their mark on the Singaporean DMD community as ...

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 ...

For columnist Patrick Moeschen, a teacher and wheelchair user, each school year is an opportunity for teaching students about ...