News

September 6, 2017

Gene Therapy for OPMD Nears Human Studies, Benitec Announces

A single gene therapy that silences the mutation responsible for oculopharyngeal muscular dystrophy (OPMD) and replaces the mutated gene with a normal one may advance into human studies in ... Read more

September 5, 2017

Upcoming Duchenne Gene Therapy Trial to Be Focus of PPMD-hosted Webinar on Wednesday, Sept. 6

News

Parent Project Muscular Dystrophy (PPMD) will host a one-hour webinar at 2 p.m. Eastern Time on Wednesday, Sept. 6, that will focus on an upcoming clinical trial exploring gene therapy for ... Read more

September 1, 2017September 1, 2017

Aberration in RNA Processing Leads to Development of Congenital Myotonic Dystrophy, Study Finds

News

In what could be the first step on the long road to finding a potential cure, researchers have found that aberrations in RNA processing are the major cause of ... Read more

August 31, 2017

Exoskeleton-supported Training Improved Movement in Limb-Girdle Patients, Small Study Found

News

Treadmill training using a voluntary-driven exoskeleton might be a safe approach to improve movement and exercise capacity in patients with limb-girdle muscular dystrophy, according to a small study by ... Read more

August 30, 2017August 30, 2017

MRI Spots Progression of Facioscapulohumeral Muscular Dystrophy Better Than Standard Methods, Study Reports

News

MRIs can pick up subtle muscle changes that standard tests cannot in slowly progressing disorders such as facioscapulohumeral muscular dystrophy, or FSHD, a study reports. The research in the journal ... Read more

August 29, 2017

Chemical Differences Made One Duchenne Therapy, Exondys 51, Succeed and Another Fail

News

The chemical structure of a drug can make the entire difference between a success and a failure in drug development. Of two drugs that used the same approach to ... Read more

August 28, 2017August 28, 2017

Researchers Isolate ST2 Protein as Potential Biomarker for Heart Disease in Duchenne Muscular Dystrophy

News

Researchers have found Duchenne muscular dystrophy (DMD) patients with heart disease have very high levels of ST2, an interleukin 1 receptor-like 1 protein. That makes this molecule a potential biomarker ... Read more

August 25, 2017

Dystrophin Protein Essential For Maturation of Brain Cells, Study Finds

News

Researchers, using a mouse model of Duchenne muscular dystrophy (DMD), have discovered that a specific protein, dystrophin, plays an essential role in myelination during postnatal brain development. The protein ... Read more

August 24, 2017August 24, 2017

PTC Therapeutics Highlights Duchenne Therapies Translarna and Emflaza in Corporate Report

News

Successful results of Translarna (ataluren) and Emflaza (deflazacort) therapies for Duchenne muscular dystrophy (DMD) are highlighted in the 2017 second quarter report by PTC Therapeutics. Orally-administered Translarna is for Duchenne ... Read more

August 23, 2017August 23, 2017

Synergy, MDA Jointly Develop Guide for Muscular Dystrophy Patients and Caregivers

News

Synergy HomeCare and the nonprofit Muscular Dystrophy Association (MDA) have teamed up in a national partnership to improve quality of life and give more independence to people with neuromuscular disorders. The ... Read more