News

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…

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…

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…

Summit Therapeutics has completed patient enrollment for PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid to treat Duchenne muscular dystrophy (DMD). PhaseOut DMD (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 (DMD), 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 (DMD), 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…

Acceleron Pharma said the first patient has been dosed in a Phase 2 clinical trial of ACE-083, its proprietary lead candidate drug ACE-083 to treat facioscapulohumeral muscular dystrophy (FSHD) — a genetic disease affecting 19,000 Americans for which no approved therapy yet exists. “ACE-083 has generated a strong data set in…

PTC Therapeutics announced that Emflaza (deflazacort), an approved treatment for all Duchenne muscular dystrophy (DMD) patients regardless of mutation, will be available in a matter of weeks in the U.S., and this time around with a starting list price of $35,000 a year — for a child weighing 25 kilograms, or about…