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).
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]
EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

PTC Therapeutics, Inc. today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion regarding the company's application for a conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older. [More]
Sildenafil may help boys suffering from muscular dystrophy disease

Sildenafil may help boys suffering from muscular dystrophy disease

Drugs used to treat erectile dysfunction may be able to slow disease progression in boys who suffer from the muscle wasting disease Duchenne muscular dystrophy. [More]
Commonly used drug restores blood flow to oxygen-starved muscles of boys with muscular dystrophy

Commonly used drug restores blood flow to oxygen-starved muscles of boys with muscular dystrophy

Cedars-Sinai Heart Institute researchers have found that a commonly prescribed drug restores blood flow to oxygen-starved muscles of boys with Duchenne muscular dystrophy, a genetic muscle-wasting disease that rarely is seen in girls but affects one in 3,500 male babies, profoundly shortening life expectancy. It is the most common fatal disease that affects children. [More]
Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

A drug typically prescribed for erectile dysfunction or increased pressure in the arteries may help improve blood flow in the muscles of boys with Duchenne muscular dystrophy, according to a study published in the May 7, 2014, online issue of Neurology®, the medical journal of the American Academy of Neurology. [More]

Race to Yes campaign lauds Sarepta's plans to seek FDA approval for Duchenne treatment

The Race to Yes campaign today lauded drug maker Sarepta on its plans to move forward immediately to seek FDA approval of the first drug to successfully treat Duchenne muscular dystrophy, the world's leading genetic killer of children. [More]