For older boys with Duchenne muscular dystrophy (DMD), 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…
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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 (DMD) and funds to advance research to further the mission of CareDuchenne, which is to find a cure…
Summit Therapeutics plc announced the publication of a study into new imaging techniques that appear to reliably and reproducibly measure utrophin protein levels and muscle fiber regeneration in muscle biopsies in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) patients. The study is important to the company because one of its…
PTC Therapeutics has announced it will submit the results its Phase 3 ACT DMD clinical trial for review by Health Canada as part of the New Drug Submission (NDS) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). DMD is a progressive disease that mostly affects male patients…
Researchers at MDI Biological Laboratory in Maine have discovered new mechanisms in heart tissue regeneration. The findings, published in the journal Development, might lead the way to drugs that help the body build new muscle cells, a crucial step toward the treatment of muscular dystrophies. Assistant Professor Voot P. Yin, who led…
Congenica, a global leader in the development of genome-based diagnostic technologies, has taken on a new project: to help a woman named Jill Viles with a serious muscle-wasting disease linked to a muscle-bound phenotype by offering to sequence her and her family’s genome. Viles and her family are affected by…
Recent advances in gene-editing technologies allow for precise manipulation of the genome to achieve therapeutic effects. While challenges remain, genome editing is likely to one day transform the treatment of muscular dystrophies, according to a review, by scientists from Editas Medicine and Duke University, that highlights current advances…
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Eligible Duchenne MD Patients to Have Expanded Access to Marathon’s Investigational Drug Deflazacort
Northbrook, Illinois-based Marathon Pharmaceuticals, a biopharma that focuses on the development of medicines to treat rare diseases with no current treatment options, has announced it is expanding patient access to its investigational medication deflazacort through its ACCESS DMD program. The program will allow participating physicians to obtain and prescribe deflazacort, which is…
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Professor’s Muscular Dystrophy Research to Be Funded with Javits Neuroscience Investigator Award
Charles Thornton, M.D., the Saunders Family Distinguished Professor in Neuromuscular Research at the University of Rochester Medical Center’s (URMC) Center for Neural Development & Disease in Rochester, New York, has received a Javits Neuroscience Investigator Award from the National Institutes of Health’s National Institute of Neurological Disorders and…
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