An experimental therapy called BIIB100 improved motor function and slowed the progression of Duchenne muscular dystrophy (Â鶹Éç) in animal models of the disease, a study shows. These findings suggest BIIB100’s potential for people with Â鶹Éç, which the researchers hope will be evaluated in a future Phase 1 trial.
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Parent Project Muscular Dystrophy (PPMD) has awarded a total of $148,000 to researchers at Nationwide Children’s Hospital and the University of Missouri to help better understand and improve the treatment of cardiac disease in people with Duchenne muscular dystrophy (Â鶹Éç). The funding falls under PPMD’s…
Treatment with Translarna (ataluren) preserves lung function and muscle strength in boys with Duchenne muscular dystrophy (Â鶹Éç), real-world data show. The most recent results from the ongoing STRIDE registry — an observational study across more than 50 care centers in Europe and Israel — were presented at…
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to ReveraGen’s vamorolone for the treatment of Duchenne muscular dystrophy (Â鶹Éç). PIM designation indicates that the MHRA considers vamorolone likely to offer major therapeutic advantages for Â鶹Éç patients. The MHRA will next…
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Novel Gene Therapy Safely Prevents Muscle Deterioration in Animal Models of Â鶹Éç, Study Reports
A novel one-time gene therapy that leads to the production of a dystrophin-related protein called utrophin prevents muscle deterioration and results in muscle regeneration in animal models of Duchenne muscular dystrophy (Â鶹Éç) without triggering an immune response, a new study reports. The study, “Non-immunogenic…
Vamorolone, an experimental treatment for Duchenne muscular dystrophy (Â鶹Éç) being developed by ReveraGen Biopharma, continues to improve muscle function in Â鶹Éç patients with fewer side effects than standard corticosteroids, topline data in 23 boys in an ongoing Phase 2 trial show. These findings were detailed in a late-breaking…
To address the need in precision medicine for human biological specimens, CureDuchenne is creating a biobank to provide Duchenne muscular dystrophy (Â鶹Éç) researchers with a collection of blood and skin tissue samples. The overarching goal is to use the biorepository to fuel and advance Â鶹Éç research. The CureDuchenne Biobank…
Experimental therapy ATL1102 improved upper limb strength and function in six boys with Duchenne muscular dystrophy (Â鶹Éç) unable to walk, according to early results of an ongoing Phase 2 clinical trial. These results also indicate that the therapy has been well-tolerated with no safety concerns reported so far.
Parent Project Muscular Dystrophy (PPMD) has awarded two grants, one to further development of a gene therapy to prevent heart failure linked to Duchenne (Â鶹Éç) and Becker muscular dystrophy (BMD), and another to create better measures of treatment responses in Â鶹Éç clinical trials. PPMD, a nonprofit organization…
Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent as an oral treatment candidate for dysferlinopathy, a group of muscle diseases that includes limb-girdle muscular dystrophy (LGMD) type 2B. Edasalonexent, formerly CAT-1004, is a small molecule designed…
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