Margarida Azevedo, MSc,  —

Patients suffering from muscular dystrophy may soon be able to independently monitor their therapeutic response to medications with a revolutionary smartphone-linked device. The Acoustical Society of America invited the creators and researchers behind this device to their annual meeting last October 30, 2014 to give a…

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…

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…

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 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…

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.