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PTC Extends Translarna To Siblings of DMD Trial’s Patients

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…

#CureFSHD Online Campaign Launched By FSH Society

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…

New MDA-Supported Genetic Therapy for DMD May Work With Only One Session

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…

PPMD Hosts Muscular Dystrophy Advocacy Conference at Capitol Hill

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…

Preimplantation Genetic Screening Procedures May Reduce Genetic Diseases

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…

aTyr Pharma’s Facioscapulohumeral Muscular Dystrophy Drug Granted Orphan Drug Status in EU

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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