News

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…

PTC Therapeutics, Inc. has just announced its novel protein restoration therapy Translarna™ (ataluren) is now available to patients with Duchenne Muscular Dystrophy (DMD) in Germany, which is the first European Union country to offer the drug. The first batch of shipments are expected to roll out this week.

A new preclinical trial revealed the efficacy and IND/CTA enabling studies with ARM210/S48168. ARMGO Pharma and Servier are set to initiate clinical trials for the treatment of Duchenne Muscular Dystrophy (DMD). The companies have been working on a drug called Rycals®. Rycals targets the Ryanodine Receptor (RyR), an intracellular calcium…

Somerset, N.J. based Catalent Pharma Solutions, specialists in efficient delivery technology and development of medical and health care product solutions is collaborating with Valerion Therapeutics, LLC of Concord, Massachusetts – whose corporate focus is on bio-therapeutics for orphan genetic disease development. The alliance will join forces to develop Valerion’s…