News

EspeRare, a company focused on uncovering the potential of existing molecules to address severe therapeutic unmet needs in rare diseases, recently received European Medicines Agency (EMA) Orphan Drug Designation for their Rimeporide compound for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a rare, inherited, life-threatening disorder that…

In 2005, quarterback Vince Young led his teammates from the University of Texas to a coveted national title while the Revell family back in Austin was just discovering that their son suffered from Duchenne Muscular Dystrophy and would be confined to a wheelchair by the time he was in college. The Revell family’s story…

On May 2, 2015 Bill and Kim Procko will be hosting a skateboarding competition “Carve to CureDuchenne,” which is intended to benefit the nonprofit CureDuchenne organization that funds research projects hoping to find a cure for Duchenne muscular dystrophy and raises awareness about the disease. The fundraising event will take place at…

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…

For most of us, using a smartphone is pretty intuitive, simply requiring touch input on the display to operate the device. However, for persons with disabilities of the upper extremities that cause difficulty using their hands and fingers, smartphones are considerably less simple to manipulate. Samsung Electronics has recently announced…

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…

Santhera Pharmaceuticals announced in a press release that an international team of researchers recently published in the journal The Lancet the results of the DELOS Phase 3 trial validating the efficacy and safety of Santhera’s Raxone/Catena (idebenone) in patients with Duchenne muscular dystrophy (DMD). The study…

Japan’s Cyberdyne, Inc. has applied for marketing authorization of their HAL for Medical Use (Lower Limb Type) robot exoskeleton as a new medical device for orphan neural-muscular diseases, such as muscular dystrophy, amyotrophic lateral sclerosis (ALS) and others under Japan’s Pharmaceutical and Medical Device Law (PMDL). Under the law,…