Margarida Azevedo, MSc,  —

aTyr Pharma (“aTyr”), a biotherapeutics company involved in the discovery and clinical development of pioneering medicines for patients with severe rare diseases using its knowledge of Physiocrine biology, a newly discovered set of physiological modulator, recently announced that Resolaris has been granted Orphan Drug Designation by the U.S.

BioMarin Pharmaceutical Inc., a company that develops and commercializes biopharmaceuticals for serious diseases and medical conditions, recently announced conclusion of the continuing proposal of a New Drug Application (NDA) to the FDA for drisapersen, an investigational exon-skipping drug candidate aimed at treating the largest genetically defined subset of…

Clinical-stage biotechnology company Capricor Therapeutics, Inc. was recently awarded orphan drug designation for its investigational cell therapy CAP-1002, from the U.S. Food and Drug Administration (FDA). The agency granted the status to the company, which is working on the development of CAP-1002 to treat …

PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company that develops novel drugs to treat diabetes and cardiovascular dysfunction in patients suffering from rare diseases, recently announced positive data from a pre-clinical study for the use of PB-1046 in Duchenne Muscular Dystrophy (DMD) mice models. The results were presented in…

Clinical stage startup company Milo Biotechnology has begun treating the first Duchenne muscular dystrophy (DMD) patients with follistatin gene therapy. The treatment is being provided through intramuscular injections and its effects in maintaining or restoring muscle function are being studied at Nationwide Children’s Hospital. The research team, which…

According to recent study, progressive myocardial fibrosis is associated with a decline in the left ventricular function in patients with Duchenne muscular dystrophy. Longer steroid treatment duration is also associated with a lower age‐related increase in myocardial fibrosis burden, according to the study now published in the journal…

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…

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…

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’…