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).

Non-Invasive prenatal blood test to detect chromosomal abnormalities in foetus

Researchers in The Netherlands believe they are on the verge of developing a simple, prenatal blood test that would be able to detect accurately chromosomal abnormalities in the developing foetus. At present, the only reliable way to do this is through amniocentesis or chorionic villus sampling, both of which are invasive and carry the risk of triggering a miscarriage.

29 Jun 2010

Prosensa, GSK initiate additional programs for DMD

Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics, announces the initiation of two further programmes under its existing alliance with GSK covering novel RNA-based treatments for Duchenne Muscular Dystrophy (DMD). The initiation of these additional programs under the terms of the existing alliance agreement is a validation of both the potential of Prosensa's "exon skipping" platform and the ongoing relationship.

23 Jun 2010

Researchers identify promising gene therapy for DMD

CureDuchenne, the Orange County non-profit focused on finding a cure for Duchenne muscular dystrophy (DMD) is one step closer to that goal through the work of Dr. Jeff Chamberlain.

23 Jun 2010

Beneficial effects of LifeVantage's Protandim on DMD mouse model published in Journal of Dietary Supplements

LifeVantage Corporation, the maker of Protandim®, a patented, science-based solution to oxidative stress, announced today that a peer-reviewed manuscript was published in the Journal of Dietary Supplements examining the beneficial effects of Protandim® on a mouse model of Duchenne Muscular Dystrophy (DMD), an X-linked inherited disease affecting one in 3,500 males.

22 Jun 2010

End Duchenne Day to be celebrated on June 26, 2010: PPMD

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy, announced that the Mayor of Denver, Colorado, John W. Hickenlooper, has proclaimed June 26, 2010 "End Duchenne Day" in recognition of PPMD's 16th Annual Connect Conference. Denver is playing host to the country's largest Duchenne-specific, international conference, June 24 – 27, 2010.

19 Jun 2010

CureDuchenne kicks off national fundraiser Pick Your Peak

CureDuchenne, a national 501(c)3 dedicated to raising funds and awareness in the fight against Duchenne Muscular Dystrophy, is kicking off a national fundraiser Pick Your Peak. Outdoor enthusiasts, philanthropists, families of boys with DMD, and anyone excited to get out and climb for a cause will climb the peak of their choice on June 19-20, 2010.

19 Jun 2010

PPMD to fund two promising research initiatives in Duchenne muscular dystrophy

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced today that PPMD will be funding two critical, promising research initiatives in Duchenne.

11 Jun 2010

Researchers underscore role of jumping genes in maintaining genetic diversity

Using high-throughput sequencing to map the locations of a common type of jumping gene within a person's entire genome, researchers at the University of Pennsylvania School of Medicine found extensive variation in these locations among the individuals they studied, further underscoring the role of these errant genes in maintaining genetic diversity.

4 Jun 2010

AMT reports positive preclinical data from cholesterol targeting AAV gene therapy study

Amsterdam Molecular Therapeutics a world leader in gene therapy, today reported positive preclinical data from a study using an AAV gene therapy product to lower cholesterol.

21 May 2010

Duchenne Muscular Dystrophy Awareness Week declared to honor work of PPMD

The Pennsylvania House of Representatives has passed a resolution declaring the week of May 24, 2010 as "Duchenne Muscular Dystrophy Awareness Week" to help honor the work of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy.

15 May 2010

NIAMS, NINDS announce 5-year, $7.5M natural history study of Duchenne muscular dystrophy

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the National Institute of Neurological Disorders and Stroke (NINDS) are launching a five-year, $7.5 million natural history study of Duchenne muscular dystrophy (Duchenne), a degenerative genetically-linked neuromuscular disease. The study aims to validate non-invasive approaches to monitor the progression and treatment of Duchenne, and holds potential to facilitate the development of promising new therapies for people with the disease.

10 May 2010

NIH awards UF scientists $7.5M for study of MRI technology in evaluating children with DMD

Duchenne muscular dystrophy research at the University of Florida got a major boost with the award of $7.5 million in National Institutes of Health funding to study the use of magnetic resonance imaging in determining the natural progression of the disease.

10 May 2010

Movements of satellite cells could help patients with muscular dystrophy: MU researchers

When muscle tissue experiences trauma or disease, such as muscular dystrophy, stem cells in the muscle known as "satellite cells" respond to repair and regenerate the muscle. These cells are particularly important in neuromuscular diseases, such as muscular dystrophy, which affect muscle stability and repair.

7 May 2010

Acceleron Pharma commences Phase 2 clinical trial of ACE-031 protein therapeutic in DMD patients

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the initiation of a Phase 2 clinical trial with ACE-031 in patients with Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease. ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling of a cell surface receptor called the activin receptor type IIB (ActRIIB).

5 May 2010

Pharmacological suppression of nonsense mutations effective in treating genetic defects

Genetic mutation can disrupt the way human cells make proteins, which in turn leads to inherited disease. David Bedwell, a professor in the University of Alabama at Birmingham (UAB) Department of Microbiology, says scientists are closer than ever to producing drugs that fix this disrupted-protein pathway and drastically improving treatment of genetic disease.

27 Apr 2010

Scientists now closer to produce drugs to fix disease-causing mutations, spare healthy genes

Inherited diseases such as cystic fibrosis can be caused by genetic "nonsense mutations" that disrupt the way human cells make proteins. David Bedwell, Ph.D., a professor in the University of Alabama at Birmingham Department of Microbiology, says scientists are now closer to producing drugs that will fix this disruption and drastically improve treatment of genetic disease.

27 Apr 2010

BioMarin Pharmaceutical introduces Firdapse for LEMS in the E.U.

BioMarin Pharmaceutical Inc. announced today that Firdapse™ (3,4-diaminopyridine) is now commercially available in the European Union (E.U.) for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). Launching immediately in Germany and the UK, the company expects to subsequently launch Firdapse in all major European markets by the end of 2010.

19 Apr 2010

New therapeutic approach for Duchenne muscular dystrophy

Researchers from Universit- Laval's Faculty of Medicine and the CHUQ Research Center have proven that it is possible to repair the defective gene responsible for Duchenne muscular dystrophy.

16 Apr 2010

New seminar series "Physical Care of Duchenne" unveiled

Duchenne Therapy Network today unveiled a new seminar series called, "Physical Care of Duchenne." The course, which includes two separate sessions taught by Jennifer Wallace, PT, will be held at the Ayres Hotel in Costa Mesa.

7 Apr 2010

Muscular dystrophy: $1M grant for study of erectile dysfunction drugs in improving muscle blood flow

A Cedars-Sinai Heart Institute cardiologist has been awarded a three-year, nearly $1 million grant from the Muscular Dystrophy Association (MDA) to study whether drugs used to treat erectile dysfunction could also be used to improve muscle blood flow and reduce fatigue in muscular dystrophy patients.

26 Mar 2010