Margarida Azevedo, MSc,  —

A European Medicines Agency (EMA) committee is beginning a review of  Sarepta Therapeutics‘ request for conditional approval of eteplirsen as a treatment for Duchenne muscular dystrophy (DMD) patients with a confirmed mutation of the dystrophin gene that can be overcome by exon 51 skipping. The Committee for Human Medicinal Products (CHMP) validated a Marketing Authorization application (MAA)…

Patients with Duchenne muscular dystrophy and Becker muscular dystrophy treated with ACE inhibitors inhibitors (drugs that dilate blood vessels) had slower progression of myocardial fibrosis than those treated with usual care. The study with the findings, “Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial,” is…

The U.K. charity Hope 4 Kidz has announced the successful completion of Matthew’s Milestone, a £75,000 (about $95,000) campaign to help a young boy with Duchenne’s muscular dystrophy (DMD) and his family. Matthew Brettel, an 11-year-old from Thorney Close, in northeast England, was diagnosed with DMD in 2013 and has been virtually wheelchair-bound since. Hope 4 Kidz,…

The Muscular Dystrophy Association (MDA) and Jiffy Lube service centers have been partners in the annual Muscle Up! campaign since 2012, joining forces to help people with muscular dystrophy (MD) live longer and grow stronger. On its 5th anniversary, the organizations are announcing that more than $5…

Exondys 51 (eteplirsen) was approved by the U.S. FDA for the treatment of Duchenne muscular dystrophy (DMD) on Sept. 19, but the drug helps only 13 percent of those battling the disease. Now, CureDuchenne is redoubling its efforts to find treatments for the 87 percent of DMD…

Summit Therapeutics has enrolled its first U.S. patients in its proof-of-concept Phase 2 clinical trial of ezutromid in patients with Duchenne muscular dystrophy (DMD). The study is actively recruiting patients. “Ezutromid has shown great promise in preclinical testing as a universal treatment that has the potential to slow…

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) favors the renewal of the conditional marketing authorization of PTC Therapeutics’ Translarna (ataluren), an investigational drug in development for ambulatory patients 5 years and older, who have nonsense mutation Duchenne muscular dystrophy…

Actelion and ReveraGen BioPharma have entered an agreement in which Actelion has obtained the exclusive option to in-license ReveraGen’s lead compound vamorolone (VBP15) for the treatment of Duchenne muscular dystrophy (DMD) at two different development stages. Vamorolone is a novel compound with the potential to preserve muscle function…

Parent Project Muscular Dystrophy (PPMD) has designated the Center for Duchenne Muscular Dystrophy (CDMD) at UCLA its 12th Certified Duchenne Care Center. PPMD is a nonprofit that works to serve the Duchenne muscular dystrophy (DMD) community through researching new treatments and demanding the best care for patients. Its…

The Assistance Fund has announced the opening of a new service, called the Duchenne Muscular Dystrophy Financial Assistance Program, to help to people living with Duchenne muscular dystrophy (DMD) meet their medical expenses. The Assistance Fund is an independent charitable patient assistance foundation that helps patients and families with  such expenses as co-payments and…