News

Newport Beach-based charity Coalition Duchenne recently launched an interview series titled “Making a Difference in Duchenne” through its Youtube channel featuring those who are making a substantial difference in Duchenne muscular dystrophy care, research, education and awareness. Dr. Eduardo Marbán, who is the director of the Cedars-Sinai Heart Institute in Los Angeles, is the…

Sarepta Therapeutics has appointed Edward Kaye, MD, as its new interim Chief Executive Officer (CEO) to lead the  effort to develop their lead product candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD). Serving as CMO since June, 2011, Kaye was in charge of the medical and…

Catabasis, a Cambridge biotech startup, is currently advancing a platform to simultaneously modulate multiple targets in disease pathways. Researchers there are especially focused on diseases like Duchenne muscular dystrophy (DMD) and Amyotrophic lateral sclerosis (ALS). The startup is now in the middle of a fundraise, striving to reach $20.4 million, according…

In a clinical trial titled “…

In a recent study titled “Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes,” a research team from the Department of Pediatric Neurology at the Catholic University in Rome, Italy examined the changes at 3 years follow-up in a 6-minute walk test…

The Muscular Dystrophy Association (MDA) recently announced that MDA clinics are offering genetic testing for those with limb-girdle muscular dystrophy (LGMD) thanks to a grant from Sanofi company Genzyme in collaboration with Emory Genetics Laboratory. This represents a major step in diagnostic advances that will help those with the disease and their respective clinicians so they can find…

Patients in Indiana who suffer from terminal diseases such as muscular dystrophy may now have access to experimental drugs that have not yet been approved by the U.S. Food and Drug Administration (FDA), but are thought to be beneficial in reducing the burden of the disease or improving patients’…

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to naproxcinod, an investigational treatment being developed by Nicox S.A. for patients with Duchenne muscular dystrophy (DMD). The designation is given to drugs or biological products that address rare conditions and is meant…

A recent study published in the journal Disease Models & Mechanisms uncovered important underlying molecular mechanisms associated with cardiomyopathy in Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy (DMD) is caused by mutations in the dystrophin gene (DMD), and characterized by progressive weakness in skeletal and cardiac muscles.

PhaseBio Pharmaceuticals, Inc., a leading company in biopolymer-based drugs developing treatments to address metabolic and specific cardiopulmonary disorders, recently announced that a $40 million financing round led by AstraZeneca has been closed. This investment from AstraZeneca, Hatteras Venture Partners, New Enterprise Associates and Johnson & Johnson Innovation – JJDC and Fletcher Spaght Ventures will serve…