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.
Findings from a recent case report published online in the journal Respiratory Medicine Case Reports suggest that patients with Facioscapulohumeral muscular dystrophy (FSHD) with nocturnal hypoventilation should have their diaphragm assessed, because nocturnal hypoventilation can be treated effectively by non-invasive ventilation. Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal…
Israeli biotech company BioBlast Pharma Ltd. has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application and is now authorized to advance development of lead product Cabaletta. The chemical chaperone is expected to become a therapeutic option for the…
The Muscular Dystrophy Association’s president and CEO opened its 2015 Scientific Conference in Washington this week by underscoring the organization’s main focus to accelerate discoveries of Duchenne muscular dystrophy (DMD) treatments for the families they serve. Steven M. Derks stated in his keynote address that the MDA has an audacious five-year plan for…
Real estate franchiser ERA is launching the ERA MDA Summer Camp Challenge, a campaign in collaboration with the Muscular Dystrophy Association (MDA) that will gather support from brokers and agents to help 1,000 children who suffer from muscular dystrophy. This year marks the fundraiser’s third year. The challenge, announced…
Biopharmaceutical company PTC Therapeutics, Inc. is not only developing a protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD), but will also expand the drug’s current developmental trial to include siblings of the patients already enrolled in their open label PTC clinical trials. Translarna will be provided…
The Muscular Dystrophy Association (MDA) is gathering the support of local communities throughout the country for another season of the MDA Muscle Walk. During this annual event, thousands of people come together to help raise both funding and awareness to help improve the quality of life of patients…
Over 150 advocates associated with the organization Parent Project Muscular Dystrophy (PPMD) gathered on Capitol Hill for PPMD’s Annual Advocacy Conference in the hope of helping accelerate the creation of federal policies to develop and provide treatment options for Duchenne muscular dystrophy (DMD). The meeting between the advocates and more…
One of the largest networks of fertility experts in California, Advanced Reproductive Care (ARC) Fertility, is advocating for the performance of new preimplantation genetic diagnosis (PGD) techniques and preimplantation genetic screening (PGS). The physicians within the network believe the procedures can help more families successfully benefit from fertility treatments and reduce the probability of their unborn…
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