News

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, Translarna™ (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…

NextCode Health Platform Will Be Used By Claritas Genomics And Might Improve Duchenne Muscular Dystrophy Diagnosis

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…

Muscular Dystrophy Association, ALS Association Collaborate To Advance Amyotrophic Lateral Sclerosis Therapy

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…

Akashi Therapeutics, Inc. To Receive $500,000 Grant From Parent Project Muscular Dystrophy To Assess HT-100

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…