The glucocorticoid deflazacort might shortly be approved for treatment of Duchenne muscular dystrophy (Â鶹Éç) 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 Â鶹Éç) for patients with Duchenne muscular dystrophy (Â鶹Éç). 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 (Â鶹Éç) 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 (Â鶹Éç). The study, “Twenty-Year Follow-Up of Newborn Screening for Patients with Muscular Dystrophy,†was published in the journal…
For older boys with Duchenne muscular dystrophy (Â鶹Éç), the time it takes to rise from the floor is associated with disease progression over 12 months, a new study reports. The findings, published in the journal PLOS ONE, indicate that the measure can be used as a prognostic factor…
Los Angeles-based Capricor Therapeutics has been awarded nearly $3.4 million to support its Phase 1/2 HOPE-Duchenne clinical trial. The grant is from CIRM — the California Institute for Regenerative Medicine — as part of the CIRM 2.0 program, a joint initiative meant to advance the development of treatments based on stem cells for people…
On April 9, hundreds of Texans will meet at the University of Texas Golf Club in Austin for the seventh annual Champions to CureDuchenne gala to raise both awareness for Duchenne muscular dystrophy (Â鶹Éç) and funds to advance research to further the mission of CareDuchenne, which is to find a cure…
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