While current studies in Duchenne muscular dystrophy (DMD) mainly explore gene and exon-skipping therapies, other options might exist in drugs already on the market. A study, published in the journal Rare Diseases, shows that the cholesterol-lowering drug simvastatin is as effective in improving muscle health as the best genetically based experimental…
Current drug development approaches for muscular dystrophy have focused mainly on loss of walking ability and do little for heart dysfunction, which often lead to premature death in patients suffering Duchenne and Becker muscular dystrophies (DMD and BMD). Attempts to develop therapies targeting heart dysfunction are underway, however, with clinical trials…
Mice lacking the immunomodulatory protein osteopontin have fewer Duchenne muscular dystrophy (DMD) symptoms, a finding researchers attributed to a switch in macrophage subsets. The study supports the notion that DMD patients could benefit from treatment with osteopontin blockers. Scientists know that immune cells, recruited to damaged muscles to initiate repair, ultimately end…
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…
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…
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…
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…
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…
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…
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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