Myotonic muscular dystrophy type 1 is a chronic and slowly progressing genetic disease affecting many organs. It is the most frequent form of muscular dystrophy and, as the name implies, is characterized by wasting of the muscles throughout the body. Nevertheless, the genetic defect on the myotonic dystrophy protein kinase also…
Grace Century’s bio banking project, Provia Laboratories, LLC, a healthcare services company specializing in high-quality bio-banking (the collection, transport, processing and cryogenic storage of biological specimens), recently reported 47% quarterly growth in enrollments for their Store-a-Tooth™ cryogenic storage service of dental stem cells. Store-A-Tooth is committed to offering…
Akashi Therapeutics Inc. a biopharmaceutical company focused on developing treatments for patients with Duchenne muscular dystrophy (DMD) recently announced encouraging interim results of its ongoing Phase 1b/2a clinical trial of HT-100 (delayed-release halofuginone) an orally available small molecule to reduce inflammation and fibrosis while promoting the regeneration of…
Non-profit organization Parent Project Muscular Dystrophy (PPMD) has nominated Duke Children’s Hospital & Health Center as a Certified Duchenne Care Center, a designation that confirms the center’s quality in the treatment of Duchenne muscular dystrophy. Duke Children’s is now the ninth center in the country to be…
The U.S. Food and Drug Administration (FDA) granted orphan drug status to an investigational therapy being developed by MediciNova, Inc. to treat Krabbe disease. The company expects MN-166 (ibudilast) to become a treatment option for the rare genetic degenerative condition that affects children at early age, causing the weakening of…
In a recent study published in Oncotarget, researchers found an inverse correlation between the level of muscle fibrosis, utrophin and the number of revertant myofibers in Duchenne Muscular Dystrophy (DMD). Results from this study reveal common links between the fibrotic and utrophin-synthesis pathways and offer new insights into…
Managing muscular dystrophy (MD) is not an easy task, and it depends not only medical intervention, but also on methods to improve patients’ quality of life. “Supporting People & Pets through Opportunity and Training” (SPPOT) is a community interest company focused on that purpose, helping to train…
Germany’s Federal Joint Committee (G-BA) published a positive Benefit Assessment for Translarna (ataluren), including an evaluation of the benefits of the drug in treating nonsense mutation Duchenne Muscular Dystrophy (nmDMD) in ambulatory patients older than five years. The therapy, which was developed by PTC Therapeutics, was rated “three” — or minor additional benefits…
A chain of convenience stores has joined forces with the Muscular Dystrophy Association (MDA) to help improve the quality of life of pediatric patients suffering from muscular dystrophy. Over 260 Western Refining convenience stores have joined the campaign to raise funding to allow more children to participate in the MDA…
EspeRare, a company focused on uncovering the potential of existing molecules to address severe therapeutic unmet needs in rare diseases, recently received European Medicines Agency (EMA) Orphan Drug Designation for their Rimeporide compound for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a rare, inherited, life-threatening disorder that…
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