Deflazacort delays loss of ambulation (LOA) in Duchenne muscular dystrophy (Â鶹Éç) 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 Â鶹Éç clinical trial (NCT01826487). The goal…
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JAR of Hope, a foundation that funds Duchenne muscular dystrophy (Â鶹Éç) 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 (Â鶹Éç) drug, Raxone (idebenone). In June 2016, the European Medicines Agency’s (EMA) CHMP validated a marketing authorization application (MAA) for Raxone…
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 (Â鶹Éç). The company expects to start enrolling patients for a placebo-controlled clinical trial of intravenous CAP-1002 in Â鶹Éç in the second…
CureDuchenne has partnered with the Muscular Dystrophy Organization Nepal (MDON) to help deliver better care to Duchenne muscular dystrophy (Â鶹Éç) patients in that country, and education and support for their families. According to CureDuchenne, Â鶹Éç affects over 300,000 children globally. An article, “Lived Experience of Mothers with Duchenne…
Levels of a protein called creatine kinase (CK) are a valuable biomarker for advanced Duchenne muscular dystrophy (Â鶹Éç), 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…
Summit Therapeutics has completed patient enrollment for PhaseOut Â鶹Éç, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid to treat Duchenne muscular dystrophy (Â鶹Éç). PhaseOut Â鶹Éç (NCT02858362) aims to provide proof-of-concept for ezutromid through measuring certain endpoints related to muscle structure, health and function. The trial could potentially provide…
Increasing the levels of a muscle-inflammation-reducing hormone called adiponectin could help reverse the progression of Duchenne muscular dystrophy (Â鶹Éç), a study suggests. The article in the American Journal of Pathology was titled “Potential Therapeutic Action of Adiponectin in Duchenne Muscular Dystrophy.†Adiponectin can reduce acute and chronic muscle inflammation. Previous…
Solid Biosciences’ SGT-001 improved muscle activity in dogs with Duchenne muscular dystrophy (Â鶹Éç), according to research the company presented at a Washington conference. Another finding of the two preclinical-trial studies was that SGT-001 increased the expression of microdystrophin, a form of the protein dystrophin found in muscle. Too little of…
Using glucocorticoid steroids once a week instead of once a day to slow the progression of muscular dystrophy (MD) could prevent downsides of the treatment, including accelerated muscle wasting, a study reports. Researchers at the Feinberg School of Medicine at Northwestern University discovered that mice with Duchenne muscular dystrophy fared better…
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