News

Santhera Pharmaceuticals has entered into a collaboration agreement with researchers from the Biozentrum of the University of Basel in Switzerland to develop a new gene therapy for treating people with LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). LAMA2 MD, also known as merosin-deficient congenital…

The Institute for Clinical and Economic Review (ICER) issued a draft report raising serious concerns about the cost-effectiveness of Sarepta‘s exon-skipping therapies Exondys 51 (eteplirsen), and the investigational golodirsen, as well as the corticosteroid Emflaza (deflazacort; by PTC Therapeutics), for treating…

Santhera Pharmaceuticals has submitted a marketing authorization request to the European Medicines Agency (EMA) for Puldysa (idebenone) as a treatment for respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD). The request is made as a conditional marketing authorization (CMA), which may be granted to medicines whose benefit…

Now that the U.S. Food and Drug Administration (FDA) has approved Zolgensma — the world’s first gene therapy for spinal muscular atrophy (SMA) — experts say a similar gene therapy to cure Duchenne muscular dystrophy isn’t far behind. On May 24, the FDA gave its long-awaited…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

A FinancialForce platform has allowed the Muscular Dystrophy Association (MDA) to enhance fundraising efforts while streamlining accounting activities. Since completing the platform launch and implementing an administrative realignment, the MDA has 40% more time to focus on fundraising strategy, according to a FinancialForce press release. Divisions such…

The Parent Project Muscular Dystrophy (PPMD) has awarded a $100,000 grant to a UCLA professor to support research on the signature profile of immune cells in patients with Duchenne and Becker muscular dystrophy. “Understanding the immune profile and how it changes through the course of disease can provide…

Sarepta Therapeutics is acquiring an investigational gene therapy program focused on calpain-3 for the treatment of limb-girdle muscular dystrophy type 2A (LGMD2A). The candidate therapy was developed by the Research Institute at Nationwide Children’s Hospital. It is Sarepta’s sixth gene therapy candidate for limb-girdle MD, which causes…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…

Joey Chorzewski has Duchenne muscular dystrophy, a neuromuscular disease that makes him different from most of his peers in Marshfield, Massachusetts. But in other ways — like wanting to wear the same kind of clothing his friends wear — he’s a typical teenager. Thanks in part to the nonprofit …