News

Duchenne Muscular Dystrophy New Drug Application Submitted to the FDA for Translarna

PTC Therapeutics, Inc. submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarnaâ„¢. The medication is used for treating nonsense mutation Duchenne muscular dystrophy (nmDMD). Duchenne muscular dystrophy (DMD) is a progressive disorder of the muscles that mostly affects males. It is caused by a…

Combined Cardiac Drugs Can Help Slow DMD Heart Failure

A study led by a cardiologist at The Ohio State University Wexner Medical Center found that administering medication for heart failure as early as possible can help slow progressive decline in cardiac function, commonly observed in young boys diagnosed with Duchenne muscular dystrophy (DMD). Lead author Dr.

Combination Medication Decreases Heart Decline in Muscular Dystrophy

Researchers at the Ohio State University Wexner Medical Center, along with experts at several other medical facilities, have found that Duchenne muscular dystrophy (DMD)-related declines in heart functioning may be slowed by already existing heart medications. The research appeared in the online version of The Lancet Neurology. DMD is characterized by…

Translarna for Mucopolysaccharidosis Granted US and EMA Orphan Drug Designation

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, Translarnaâ„¢ (ataluren), has received orphan-drug designation from the US Food and Drug Administration and European Medicines…