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…
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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…
The effectiveness of Deflazacort (DFZ) against prednisone was tested in Duchenne muscular dystrophy (DMD) patients, and positive results were reported at the 2016 MDA Clinical Conference in Arlington, Virginia, in a presentation by Marathon Pharmaceuticals titled “Effect of Deflazacort and Prednisone on Muscle Enzymes in the Treatment of Duchenne Muscular…
The glucocorticoid deflazacort might shortly be approved for treatment of Duchenne muscular dystrophy (DMD) in the US. In preparation for the approval process, Marathon Pharmaceuticals have released new data showing that in young rats, the drug causes a slowing of growth but is otherwise safe. Deflazacort has been used…
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Duchenne MD Patients Now Have Expanded Access to Marathon’s Deflazacort Investigational Treatment
Marathon Pharmaceuticals has announced its investigational medicine deflazacort will be accessible for free through an Expanded Access Program (ACCESS DMD) for patients with Duchenne muscular dystrophy (DMD). Deflazacort will now be made available to eligible patients through a growing network of medical research centers across the nation. “In…
Researchers at Japan’s Kobe University Graduate School of Medicine have successfully decoded a sugar molecule, providing an important step toward understanding a mechanism linked to muscular dystrophy (MD). The study, “Identification of a Post-translational Modification with Ribitol-Phosphate and Its Defect in Muscular Dystrophy,” was published in the journal…
University of Nottingham researchers at the Sir Peter Mansfield Imaging Centre (SPMIC) have been awarded a more than £1 million grant to develop a magnetic resonance imaging (MRI) technique that uses the body’s natural sodium ions to provide more advanced pictures of healthy and diseased tissue. MRI is used to diagnose and…
The only drugs currently recommended for people with Duchenne muscular dystrophy (DMD) are glucocorticoid corticosteroids, but many patients still do not receive such treatment. A review in the journal US Neurology underscores the benefits of deflazacort, a glucocorticoid used as an anti-inflammatory and immunosuppressant, demonstrating that the drug…
Ultragenyx, a biopharma dedicated to the development of investigational products for rare diseases, will fund the efforts of a team of scientists from Saint Louis University (SLU) to advance a treatment for muscular dystrophy, inspired by lead researcher Dr. Fran Sverdrup’s daughter, who was diagnosed with the disease. SLU’s Center…
Researchers from the University of Pittsburgh recently released key findings from a study which may hold important implications for novel therapies designed to treat patients with Duchenne muscular dystrophy (DMD). The study, “Twenty-Year Follow-Up of Newborn Screening for Patients with Muscular Dystrophy,” was published in the journal…
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