Duchenne Muscular Dystrophy News and Research RSS Feed - Duchenne Muscular Dystrophy News and Research

Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).
Duke researchers grow contracting human skeletal muscle in lab

Duke researchers grow contracting human skeletal muscle in lab

In a laboratory first, Duke researchers have grown human skeletal muscle that contracts and responds just like native tissue to external stimuli such as electrical pulses, biochemical signals and pharmaceuticals. [More]
New way to measure upper extremity movement in muscular dystrophy patients

New way to measure upper extremity movement in muscular dystrophy patients

Researchers at Nationwide Children's Hospital have developed a way to measure upper extremity movement in patients with muscular dystrophy using interactive video game technology. Their hope is to expand inclusion criteria for clinical trials to incorporate patients using wheelchairs. [More]
Neurological testing accessibility and affordability: an interview with Dr Joseph Higgins

Neurological testing accessibility and affordability: an interview with Dr Joseph Higgins

Neurological disorders with genetic causes can be very difficult to diagnose without reliable, clinically relevant tests tailored to specific phenotypes. [More]
Early use of heart failure drugs slows cardiac damage in boys, young men with DMD

Early use of heart failure drugs slows cardiac damage in boys, young men with DMD

Early use of available heart failure drugs slows the progressive decline in heart function before symptoms are apparent in boys and young men with Duchenne muscular dystrophy (DMD), according to a new study published online by The Lancet Neurology. [More]
PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC Therapeutics, Inc. today announced that it has commenced a rolling submission of a New Drug Application to the United States Food and Drug Administration for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). [More]
Cedars-Sinai Medical Center releases November tip sheet of story ideas

Cedars-Sinai Medical Center releases November tip sheet of story ideas

Following is the November 2014 tip sheet of story ideas from Cedars-Sinai Medical Center. [More]
Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers at the Center for iPS Cell Research and Application, Kyoto University, show that induced pluripotent stem (iPS) cells can be used to correct genetic mutations that cause Duchenne muscular dystrophy (DMD). [More]
PPMD, Santhera Pharmaceuticals collaborate to study Duchenne muscular dystrophy

PPMD, Santhera Pharmaceuticals collaborate to study Duchenne muscular dystrophy

Parent Project Muscular Dystrophy, the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) and Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focusing on the development and marketing of innovative pharmaceutical products for the treatment of mitochondrial and neuromuscular diseases, will collaborate on a benefit/risk study in Duchenne. [More]
Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease. [More]
Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers from the Centre for Comparative Genomics have received almost $800,000 in funding from the National Health and Medical Research Council to develop genetic drugs to treat rare diseases. [More]
Royal Holloway-led researchers to develop novel spinal cord injury treatment

Royal Holloway-led researchers to develop novel spinal cord injury treatment

Dr Rafael Yáñez-Muñoz, from the School of Biological Sciences at Royal Holloway, University of London, is leading a team of researchers working to develop a novel treatment for spinal cord injury - which leaves sufferers with devastating, life-long effects including paralysis. [More]
State highlights: Calif. ballot measure updates; Va. prison health care budget shortfall

State highlights: Calif. ballot measure updates; Va. prison health care budget shortfall

A selection of health policy stories from California, Arizona, Maine, South Dakota, Virginia, Maryland, Louisiana, Pennsylvania, Massachusetts, North Carolina, Texas, Washington state and Missouri. [More]

MD-CARE Act Amendments become law

Capping a two-year advocacy campaign led by Parent Project Muscular Dystrophy, today President Barack Obama signed the MD-CARE Act Amendments of 2014 into law, providing a significant victory to the Duchenne and broader muscular dystrophy communities. [More]
Research offers hope to patient suffering from Duchenne muscular dystrophy

Research offers hope to patient suffering from Duchenne muscular dystrophy

Research at Stockholm's KTH Royal Institute of Technology offers hope to those who suffer from Duchenne muscular dystrophy, an incurable, debilitating disease that cuts young lives short. [More]
New gene therapy protects mice from life-threatening heart condition

New gene therapy protects mice from life-threatening heart condition

A new gene therapy developed by researchers at the University of Missouri School of Medicine has been shown to protect mice from a life-threatening heart condition caused by muscular dystrophy. [More]
Researchers use new gene editing method to correct mutation that leads to DMD

Researchers use new gene editing method to correct mutation that leads to DMD

UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition. [More]
Isoform: New protein offers novel therapeutic approach for patients with DMD

Isoform: New protein offers novel therapeutic approach for patients with DMD

Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. [More]
Splice-switching oligonucleotide drugs alter editing of gene transcript

Splice-switching oligonucleotide drugs alter editing of gene transcript

In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. [More]
PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics, Inc. today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna (ataluren) available to patients before commercial availability in certain countries. [More]
FDA grants Fast Track designation to Akashi Therapeutics' HT-100 for treatment of DMD

FDA grants Fast Track designation to Akashi Therapeutics' HT-100 for treatment of DMD

Akashi Therapeutics, Inc., announced today that the U.S. Food and Drug Administration has granted Fast Track designation to the company's most advanced product candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. [More]