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).

Gene delivery strategy to help improve muscle mass in patients with degenerative muscle disorders

Severe weakness of the quadriceps is a defining feature of several neuromuscular disorders. Researchers at Nationwide Children's Hospital have shown that a gene delivery strategy that produces follistatin - a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in skeletal muscle - directly to the quadriceps of non-human primates results in long-term gene expression with muscle enhancing effects, including larger muscles with greater strength.

12 Nov 2009

AMT's gene therapy successful in treating Duchenne muscular dystrophy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that it has successfully treated Duchenne muscular dystrophy (DMD) in an animal model with its proprietary gene therapy. The proof of concept studies were performed in collaboration with the group of Professor Irene Bozzoni (University of Rome, La Sapienza, Italy) and demonstrated effectiveness in the heart as well as in skeletal muscles.

11 Nov 2009

PPMD awards $75,000 grant to University of California professor for Duchenne muscular dystrophy research

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 the latest recipient of the End Duchenne Grant Award Program.

6 Nov 2009

Research shows exon-skipping drug may prove effective for treating Duchenne muscular dystrophy

An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan.

21 Oct 2009

Amsterdam Molecular Therapeutics' AMT-080 granted EMEA Orphan Drug Designation

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.

13 Oct 2009

Macrophages play a crucial role in muscle regeneration

For scientists at the European Molecular Biology Laboratory (EMBL) in Monterotondo, Italy, what seemed like a disappointing result turned out to be an important discovery.

22 Sep 2009

Charley's Fund and the Nash Avery Foundation to support investigation of Galapagos' SARM candidate drug

Galapagos NV (Euronext: GLPG), Charley's Fund Inc. and the Nash Avery Foundation announced today that they will collaborate to investigate the potential effectiveness of Galapagos' SARM candidate drug, G100192, in treating Duchenne muscular dystrophy. G100192 is an orally-available small molecule therapeutic, which has demonstrated successful Proof of Concept in pre-clinical studies for cachexia (the loss of weight and muscle mass).

17 Sep 2009

Safeway raises funds to support neuromuscular disease medical research and therapies

Safeway Inc. announced today it has raised a record $10.2 million to support medical research and services for the millions of people living with neuromuscular diseases. The funds were raised during Safeway’s annual Muscular Dystrophy Association (MDA) campaign, a five-week endeavor to highlight the need to find cures and treatments for muscular dystrophy and related diseases, and presented during the Jerry Lewis MDA Telethon in Las Vegas Labor Day weekend.

9 Sep 2009

AVI BioPharma's peptide-conjugated PMO chemistry to be discussed at two upcoming scientific meetings

AVI BioPharma, Inc., a developer of RNA-based drugs, today announced that Hong Moulton, Ph.D., Director of Discovery Research, will give oral presentations highlighting improved analogues of AVI's phosphorodiamidate morpholino oligomer (PMO) chemistry at two upcoming scientific meetings.

28 Aug 2009

Amsterdam Molecular Therapeutics reports 2009 half year financials

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today reported its results for the first half year of 2009.

13 Aug 2009

New function for missing protein in Duchenne muscular dystrophy found

Researchers at the University of Minnesota and National Institutes of Health have identified a new function for the protein missing in people with the most common and ultimately lethal form of childhood muscular dystrophy.

3 Aug 2009

New therapy shows potential for treating Duchenne muscular dystrophy

Researchers at the University of Minnesota Medical School have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children.

26 May 2009

Inflammation clue to fragile bones in Duchenne's muscular dystrophy

Inflammation could contribute to bone loss in Duchenne's muscular dystrophy (DMD), a discovery made by a group of Italian researchers. Dr Anna Rufo and her colleagues found that levels of an inflammatory molecule, known as IL-6, are high in patients with DMD.

25 May 2009

Muscular dystrophy diagnosis delayed in boys

Boys show signs of Duchenne Muscular Dystrophy (DMD) for 2 ½ years before they obtain a diagnosis and disease-specific treatment, about the same length of delay children have endured for the past 20 years despite advances in genetic testing and treatment.

11 May 2009

Antibiotics could treat cystic fibrosis, other genetic diseases

By modifying the properties of the common antibiotic gentamicin, researchers at the Technion-Israel Institute of Technology have developed what could become an effective treatment for many human genetic diseases, including cystic fibrosis (CF), Duchenne muscular dystrophy, Usher Syndrome and numerous cancers.

8 Apr 2009

First treatment for muscular dystrophy a step closer

Genetic researchers at Children's National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of "multiple exon-skipping" to curb the devastating effects of Duchenne muscular dystrophy in an animal larger than a mouse.

16 Mar 2009

Novel genetic technology for canine form of muscular dystrophy

Using a novel genetic technology that covers up genetic errors, researchers funded in part by the National Institutes of Health have developed a successful treatment for dogs with the canine version of Duchenne muscular dystrophy, a paralyzing, and ultimately fatal, muscle disease.

16 Mar 2009