Duchenne Muscular Dystrophy News and Research

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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).

First human clinical study of ChromaDex's NIAGEN nicotinamide riboside meets primary endpoint

ChromaDex Corp. announced today that the initial results of the first human clinical study for the company's NIAGEN nicotinamide riboside (NR) has met its primary endpoint.

11 Feb 2015

UI Children's Hospital named Certified Duchenne Care Center by PPMD

The University of Iowa Children's Hospital was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) and demanding optimal care for all people with Duchenne.

7 Feb 2015

Study demonstrates therapeutic potential of new class of synthetic oligonucleotides for DMD treatment

Research efforts associating scientists from the CNRS, UVSQ and INSERM within the Laboratoire END-ICAP, working in collaboration with a team from the University of Bern, has demonstrated the therapeutic potential of a new class of synthetic oligonucleotides in the treatment of Duchenne muscular dystrophy (DMD) using RNA "surgery".

4 Feb 2015

Bern researchers develop active substance for treating Duchenne muscular dystrophy

Duchenne muscular dystrophy is a congenital disease which causes muscle degeneration and eventual death in teenagers. Recently, researchers from Bern developed an active substance, which they together with an international team tested successfully.

3 Feb 2015

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.

13 Jan 2015

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.

12 Jan 2015

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.

From Quest Diagnostics 5 Jan 2015

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.

30 Dec 2014

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).

23 Dec 2014

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.

27 Nov 2014

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).

27 Nov 2014

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.

25 Nov 2014

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.

18 Nov 2014

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.

6 Nov 2014

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.

29 Oct 2014

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.

21 Oct 2014

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.

27 Sep 2014