News

South Plainfield, NJ-based biopharmaceutical company PTC Therapeutics, Inc. recently announced that it has launched a rolling submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for pipeline nonsense mutation Duchenne muscular dystrophy (nmDMD) treatment Translarna™. This process enables…

Stuart W. Peltz, Ph.D, the CEO of biopharmaceutical company PTC Therapeutics, Inc., was recently granted the 2014 Legend of Technology Award for Vision, Innovation, and Leadership, in recognition of the impact of his work in the field of technological industry based largely on PTC’s investigational therapy for DMD. The…

A trio of companies, comprised of CITGO Marketer Flash Market Inc., Core Mark and CITGO Petroleum Corporation recently hosted the 10th Annual Passport to a Cure benefit in Little Rock, Arkansas for the local chapter of the Muscular Dystrophy Association (MDA), the world’s leading non-profit organization for muscle disease, including muscular…

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…