News

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…

Isis and Biogen Advance ISIS-DMPKRx to Phase 1/2 Myotonic Dystrophy Type 1 Study

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…