The FDA is considering whether to conditionally approve a new treatment for DMD patients with mutations amenable to exon 51 ...
Commendamide, a metabolite produced by beneficial gut bacteria that are depleted in people with DMD, protects muscle cells ...
After two difficult years, columnist Betty's Vertin's son Max won't go back to college this fall, a decision that has her breathing easier.
CureDuchenne has launched a national public service announcement campaign to highlight the urgent need for new treatments for ...
There are 24 hours in a day, so there should be enough time in each one to keep from feeling caregiver burnout, says columnist.
Columnist Shalom Lim is grateful that his profile on the Purple Parade website highlights the importance of his disability ...
This year’s Be Their Muscle campaign has raised more than $641,000 to support the MDA Summer Camp and help advance research ...
Key milestones have been met in a clinical trial testing a one-time treatment that aims to turn off the faulty gene in FSHD, ...
While Duchenne muscular dystrophy (DMD) is one of the most common types of this group of muscle-wasting disorders, affecting about 1 in 3,500 male births, each patient’s journey with this form of ...
Exon skipping is a treatment approach for people whose Duchenne muscular dystrophy is due to certain mutations in its causative gene. Exon skipping works like a molecular patch, so that the DMD gene ...
Exon-skipping therapies Amondys 45 (casimersen) and Vyondys 53 (golodirsen) show “positive and encouraging trends” for people with Duchenne muscular dystrophy (DMD), even though top-line results from ...
Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of its cell therapy deramiocel to treat Duchenne muscular dystrophy (DMD)-related heart disease ...