PTC Therapeutics, Inc., a biopharmaceutical company with a focus on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes, has just announced one of its leading pipeline products, Translarna™ (ataluren), has received orphan-drug designation from the US Food and Drug Administration and European Medicines…
The “Solid Suit” is actually designed to be soft, like the one pictured above, and could be worn by people with DMD under their clothes. Hackensack, N.J. based nonprofit Parent Project Muscular Dystrophy (PPMD), which is dedicated to fighting and eventually prevailing over Duchenne muscular dystrophy…
According to a new research study by scientists at the Stanford University School of Medicine in mouse modeling of Duchenne muscular dystrophy, faulty connective-tissue genes associated with symptoms like fibrosis and muscle weakness are expressed in muscle stem cells of afflicted subjects. As with human patients, mice with…
Carlsbad, California-based Isis Pharmaceuticals, Inc. recently announced the launch of a study for ISIS-DMPKRx, indicated to treat Myotonic Dystrophy Type 1 (DM1), a rare neuromuscular disease caused by an abnormal and toxic production of dystrophia myotonica-protein kinase (DMPK) RNA in cells, which is an abnormally long strand of RNA, that accumulates in cell…
Claritas Genomics announced that it will be using the NextCode Health’s integrated and clinical research platform to analyze sequencing-based clinical diagnostic tests. This partnership will allow Claritas to interpret its genome analysis faster and more efficiently so that tests and results can be delivered earlier, improving diagnosis of diseases such as duchenne muscular…
The Parent Project Muscular Dystrophy (PPMD), a not-for-profit organization supporting research into Duchenne muscular dystrophy (DMD) and providing patient-family support, has just announced it has signed a partnership agreement with clinical stage biotechnology company, Catabasis Pharmaceuticals, Inc., to collaborate on a Phase II clinical study…
The ALS Association and the Muscular Dystrophy Association (MDA) recently teamed up to fund a research project whose goal is to find a therapy for amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s Disease. The nonprofit associations are both committed to funding and fostering treatments and an eventual cure for ALS, and to provide care…
The Parent Project Muscular Dystrophy (PPMD) recently petitioned Congress to ask for more support in strengthening existing programs dedicated to finding treatment options and an eventual cure for Duchenne muscular dystrophy. The association hopes to increase federal funding and awareness for patients suffering from the disease. Specifically, PPMD is…
The Parent Project Muscular Dystrophy (PPMD) organization has announced that it will award a $500,000 grant to Akashi Therapeutics, Inc. (Akashi) to fund clinical trials that will assess whether the company’s HT-100 (delayed-release halofuginone), a new investigational drug, is safe and effective against Duchenne muscular dystrophy. Duchenne muscular dystrophy…
Encouraged by the acquisition of biotechnology company Prosensa by BioMarin Pharmaceutical, CureDuchenne has announced that it will continue to support Prosensa’s drug development efforts for DMD. CureDuchenne, a national nonprofit organization that is dedicated to finding a cure and reducing patients’ burden of suffering from the disease, has been supporting Prosensa…
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