In this video from Muscular Dystrophy UK, two teenagers share what it’s like living with the disease and some of the frustrations they experience on a day-to-day basis. MORE: How service dogs can benefit people with neuromuscular disorders. Fourteen-year-old Sam from Cheltenham and 13-year-old Zoe…
Deflazacort delays loss of ambulation (LOA) in Duchenne muscular dystrophy (DMD) patients by an average of 3.8 years more than standard of care corticosteroid treatments prednisone/prednisolone, post-hoc analyses of a Phase 3 clinical trial found. The new conclusions follow a reassessment of data obtained in the placebo group of the completed ACT DMD clinical trial (NCT01826487). The goal…
JAR of Hope, a foundation that funds Duchenne muscular dystrophy (DMD) research, is working to get legislation approved to make September 10-16 Duchenne Muscular Dystrophy Awareness Week in New York state. The foundation, along with New York City Assemblyman Michael Cusick, will ask New York Gov. Andrew M. Cuomo on…
Santhera Pharmaceuticals has updated the timeline for the ongoing assessment by the European Committee for Medicinal Products for Human Use (CHMP) of its Duchenne muscular dystrophy (DMD) drug, Raxone (idebenone). In June 2016, the European Medicines Agency’s (EMA) CHMP validated a marketing authorization application (MAA) for Raxone…
A powerful new genome editing tool clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a solution in the treatment of Duchenne muscular dystrophy (DMD). CRISPR/Cas9 mimics the way bacteria works by eliminating the DNA of invading viruses and replacing it with part of its own DNA, slicing into the DNA and rebuilding…
Positive results from the HOPE-Duchenne clinical trial support further clinical development of Capricor Therapeutics’ therapy candidate CAP-1002 for treatment of cardiac anomalies caused by Duchenne Muscular Dystrophy (DMD). The company expects to start enrolling patients for a placebo-controlled clinical trial of intravenous CAP-1002 in DMD in the second…
CureDuchenne has partnered with the Muscular Dystrophy Organization Nepal (MDON) to help deliver better care to Duchenne muscular dystrophy (DMD) patients in that country, and education and support for their families. According to CureDuchenne, DMD affects over 300,000 children globally. An article, “Lived Experience of Mothers with Duchenne…
Having a service dog can greatly enhance the life of someone living with muscular dystrophy. They allow patients to regain some of their independence by helping with small everyday tasks like opening and closing doors, fetching meds, acting as a prop or support as their owner stands, switching on lights and attracting attention…
Gentle exercise has many health benefits, particularly for caregivers of patients with chronic illnesses. In this video, personal trainer Ryan Krane demonstrates some simple exercises that can help counter the effects of chronic fatigue. MORE: Watch an animated explanation of muscular dystrophy here. Krane shares five simple…
Levels of a protein called creatine kinase (CK) are a valuable biomarker for advanced Duchenne muscular dystrophy (DMD), but now researchers say it may also be a strong predictor of patients’ lung function. The study, “Correlation of Serum Creatine Kinase Level With Pulmonary Function in Duchenne Muscular Dystrophy,” was…
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