News

A new version of the CRISPR-Cas9 gene-editing technology called CRISPR-Gold has successfully restored the correct sequence of the dystrophin gene in a mouse model of Duchenne muscular dystrophy (DMD), a new study revealed. Researchers found that an injection of CRISPR-Gold into DMD mice led to an 18-times-higher correction rate and…

Mice with Duchenne muscular dystrophy compensate for the weakness in their breathing muscles by sending stronger signals to the nerves controlling the diaphragm, according to scientists at the University College Cork in Ireland. Now it is important to examine whether the same mechanisms exist in human Duchenne patients, researchers…

Orphan drug development, rising healthcare costs, patient assistance programs and controversial right-to-try legislation are of paramount interest to the parents and caregivers of boys with Duchenne muscular dystrophy (DMD). “One of the chief challenges of 21st Century pediatric medicine is our continued inability to provide more help to children born with…

Resolaris (ATYR1940) improved the muscle strength of nearly two-thirds of adolescents and young adults with early-onset facioscapulohumeral muscular dystrophy (FSHD), according to final results of a Phase 1/2 clinical trial. The therapy’s maker, aTyr Pharma, presented the findings at the 22nd International Annual Congress of the World Muscle Society (WMS) in Saint…

Parent Project Muscular Dystrophy (PPMD), one of the largest nonprofit organizations in the U.S. working to find a cure for Duchenne muscular dystrophy (DMD), recently announced that it will continue to support a grant for the Certified Duchenne Care Center (CDCC) Program. PPMD invests in research, advocacy, and initiatives to…

Capricor Therapeutics presented positive six-month results from the HOPE trial of CAP-1002, a stem cell-based therapy for Duchenne muscular dystrophy (DMD), at the 22nd International Congress of the World Muscle Society, Oct. 3-7 in St. Malo, France. The poster is available on Capricor’s website. The Phase 1/2 HOPE trial…

The drug discovery and development biopharma Summit Therapeutics is giving several presentations related to the ongoing PhaseOut DMD clinical trial at the 22nd International Congress of the World Muscle Society, Oct. 3-7 in St. Malo, France. One of the poster presentations includes baseline…

Positive results were reported by Catabasis Pharmaceuticals from the open-label extension MoveDMD clinical trial evaluating edasalonexent (CAT-1004) in boys with Duchenne muscular dystrophy (DMD). A global Phase 3 trial of the investigative drug is projected for the first half of 2018. The new data were presented at…

With one exon-skipping therapy — Exondys 51 (eteplirsen) — approved for Duchenne muscular dystrophy (DMD) and others in clinical trials, it’s easy to envision Sarepta Therapeutics as the “exon-skipping company.” But the idea of current exon-skipping treatments as the ultimate for DMD couldn’t be further from what Sarepta…

Parents of boys with Duchenne muscular dystrophy (DMD) as well as patient advocacy groups are praising the recent approval of Sarepta Therapeutics‘ Exondys 51 (eteplirsen) to treat the disease. But the decision by the U.S. Food and Drug Administration (FDA) to approve a treatment without clear data supporting…