News

Marathon Pharmaceuticals recently presented additional results from a Phase 3 clinical trial of deflazacort, an investigational glucocorticoid corticosteroid that the company hopes will be approved for the treatment of Duchenne muscular dystrophy (DMD) early next year. The data was presented at the 68th American Academy of Neurology (AAN) Annual Meeting, running through April…

Parent Project Muscular Dystrophy (PPMD) is reaching out to families affected by Duchenne muscular dystrophy (DMD) through its ‘Every Single One’ Tour, a multi-year campaign to bring updates on disease research, care, and advocacy efforts to communities across the U.S. The campaign, whose daylong sessions are free of charge, kicked…

University of Chicago researchers developed a DNA nanosensor that measures the physiological concentration of chloride with a high degree of accuracy, a discovery with critical implications for diseases such as muscular dystrophies. Until now, there was no effective way to measure intracellular stores of chloride. The newly developed sensor, called…

Parent Project Muscular Dystrophy (PPMD), the largest U.S. nonprofit group focused on Duchenne muscular dystrophy (DMD), is holding a Pulmonary Outcomes Workshop this week, April 14–15. Physicians and industry experts will discuss pulmonary outcome measures used in current DMD clinical trials, how those measures might be improved, and their use in furthering drug development.

The Muscular Dystrophy Coordinating Committee (MDCC) has released an updated version of its Action Plan for the Muscular Dystrophies, a document that works as a comprehensive guide to address the challenges experienced by people with muscular dystrophy. The committee, authorized by Congress, is composed of representatives from U.S. federal agencies and…

Researchers at Switzerland’s Basel University Hospital investigated the biochemical and physiological properties of the muscles involved in the control of the movements of the eyelids along with the extraocular muscles, which control eye movements, noting that these muscle groups express low levels of dystrophin and high levels of utrophin, a similar protein…

Deflazacort, a glucocorticoid drug, was shown to improve muscle strength in boys with Duchenne muscular dystrophy (DMD) in a clinical trial performed by Marathon Pharmaceuticals. Trial results were reported in a presentation, “Effect of DEFLAZACORT and PREDNISONE versus placebo on muscle strength in boys with duchenne muscular dystrophy who have lost ambulation: results…

Marathon Pharmaceuticals recently presented findings from a Phase 3 trial comparing the effects of deflazacort to prednisone and placebo in boys with Duchenne muscular dystrophy. The presentation, showing deflazacort was more effective than prednisone in timed functional tests and in limiting weight gain, was given at the 2016 Muscular Dystrophy Association (MDA) Clinical Conference, and will be shown again at…

Duchenne muscular dystrophy (DMD) patients at more advanced disease stages, those reliant on wheelchairs, showed improved lung function when treated with deflazacort compared to prednisone, Marathon Pharmaceuticals reported in a presentation at the recent MDA Clinical Conference in Arlington, Virginia. The presentation, “Effect of deflazacort and prednisone versus placebo on pulmonary function in boys…

Ten-year-old Gabe Griffin of Alabama visited Washington, D.C., with his parents last month hoping to encourage Congress to pass a federal bill that could help accelerate the discovery of an effective treatment or cure for the rare and terminal form of muscular dystrophy he’s afflicted with. Duchenne…