Everyone’s idea of what makes for a happy life is different. For example, some people like traveling all over the world or have a bucket list of places to go and things to do. On the other hand, some people simply want to have a good career and…
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…
This video from Muscular Dystrophy UK is all about 16-year-old Karis Williamson from Inverness, Scotland. Karis used to love going to school, but as she got older, she found it frustrating and felt like an unvalued member of the class. Feeling left out and like a burden, she…
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…
This Macmillan Cancer Support video shares some tips on how to get the most out of your doctors’ appointments and hospital visits. MORE: Comedian with muscular dystrophy discusses jokes, dating and sex Dr. Davina Deniszczyc explains that before you meet with your doctor, it’s a good idea to prepare…
The Muscular Dystrophy Association (MDA) is the largest organization in the U.S. dedicated to helping children and adults with muscular dystrophy (MD), as well as their caregivers and families. In addition to the resources it offers on a full-time basis, the MDA also has some limited-time opportunities…
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…
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