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…
A late-stage clinical trial performed by researchers at the University of Florida Health (UF Health) led to a new promising treatment for children with the most common form of muscular dystrophy, Duchenne Muscular Dystrophy. This condition causes the loss of mobility when the patients reach 12 years old, and…
The FSH Society, a charity organization based in Massachusetts supporting research and advocacy related to facioscapulohumeral muscular dystrophy (FSHD) has recently announced the launch of the #CureFSHD campaign. The aim of this campaign is to boost awareness and to educate people about FSHD, which is a type of…
Charles Gersbach, Muscular Dystrophy Association research grantee and Biomedical Engineering Professor at Duke University, has recently announced an enormous and potentially game-changing development in gene modification for young men and boys suffering from Duchenne muscular dystrophy (DMD). The results were published in Nature Communications in a…
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…
BioBlast Pharma, a clinical-stage biotechnology firm focused on developing orphan disease drugs, recently announced it has received an allowance from the United States Patent and Trademark Office (USPTO) regarding a method to address oculopharyngeal muscular dystrophy (OPMD) with Cabaletta.
Muscle wasting in muscular dystrophy may be repairable using a new approach developed by a collaboration among researchers in Italy, Israel, and the United Kingdom. Published in EMBO Molecular Medicine, a new study, “In Vivo Generation of a Mature and Functional…
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…
Biotherapeutics company aTyr Pharma, Inc. was recently awarded orphan drug designation from the European Commission (EC) for its Resolaris therapy, designed to treat facioscapulohumeral muscular dystrophy (FSHD). The investigational drug is the company’s first Physiocrine-based therapy, and is currently being developed to become a first-in-class intravenous protein treatment for…
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