Tag: Duchenne Muscular Dystrophy (DMD)

News, Research

UC Davis Researchers Seek DMD Participants for Survey to Improve Clinical Trial Outcome Measurements

MDA Staff

In order to develop better methods for capturing the perspective of individuals living with Duchenne muscular dystrophy (DMD) during clinical trials, researchers at the University of California Davis Neuromuscular Research Center are seeking participants for an online survey to monitor movement and daily living abilities. The Duchenne Muscular Dystrophy Life-Time Mobility Scale (DMD-LMS) is a . . .

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News, Living with Muscular Dystrophy

MDA, PPMD, TREAT-NMD and World Duchenne Organization Release New Duchenne Guide for Families

Robin Geiger

The first standards of care for Duchenne muscular dystrophy (DMD) were published by the U.S. Centers for Disease Control and Prevention (CDC) in 2010. And today — on World Duchenne Awareness Day 2018 — the DMD care landscape is already, and excitingly, a much different space. Several new therapies, including the first-ever disease modifying DMD . . .

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News

Sarepta Therapeutics Announces Clinical Hold of Phase 1/2a DMD Micro-Dystrophin Gene Therapy Trial

MDA Staff

Late on Wednesday, Sarepta Therapeutics announced that the Food and Drug Administration (FDA) has placed a clinical hold on their Phase 1/2a Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial due to contamination of the manufacturing lot with plasmid DNA. No safety issues were reported for participants in the trial. Sarepta has stated that they . . .

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News, Research

Summit Therapeutics Discontinues ezutromid for DMD after Disappointing Phase 2 Trial Results

MDA Staff

This morning, Summit Therapeutics announced that it is discontinuing development of ezutromid for Duchenne muscular dystrophy after primary and secondary endpoints were missed after 48 weeks of treatment in their PhaseOut DMD trial. PhaseOut DMD was a phase 2, multi-center, open-label clinical trial of ezutromid, a utrophin-modulation therapy. Thirty-eight boys enrolled in the trial completed the 48-week regimen. The . . .

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News, Research

Sarepta Reports Positive Preliminary Findings in DMD Gene Therapy Trial

MDA Staff

This week, Sarepta Therapeutics announced encouraging preliminary results from a phase 1/2a gene therapy trial designed to assess the investigational drug AAVrh74.MHCK7.micro-Dystrophin in boys with Duchenne muscular dystrophy (DMD). After 90 days in the trial, the first three participants all showed robust expression of micro-dystrophin — a shortened version of the protein that is absent . . .

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News, Research

Santhera Seeks Participants for DMD Study, Makes Available EAP and Online DMD Resource

Amy Madsen

With a focus on respiratory care in Duchenne muscular dystrophy (DMD), Santhera Pharmaceuticals has made available new resources for individuals with the disease. In addition, the company is seeking individuals to participate in a phase 3 clinical trial to test a respiratory drug in people with DMD. SIDEROS trial seeks participants Researchers are looking for . . .

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Personal Stories, Kids, Living with Muscular Dystrophy

“Strength is More than Muscle”: A Mother Reflects on a Year of Grief and Growth After Son’s DMD Diagnosis

Sarah Krieger

One year ago, if you would have asked me to say the words Duchenne muscular dystrophy, I couldn’t. It was too new, too fresh, too difficult of a reality to process. One year ago I fell to my knees crying and physically shaking with fear, anxiety and anger as I hung up the phone with . . .

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