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).

New gene therapy approach safe for patients with muscular dystrophy

Researchers at the University of North Carolina at Chapel Hill have shown that it is safe to cut and paste together different viruses in an effort to create the ultimate vehicle for gene therapy. In a phase I clinical trial, the investigators found no side effects from using a "chimeric" virus to deliver replacement genes for an essential muscle protein in patients with muscular dystrophy.

1 Dec 2011

Critical breakthrough in development of treatment for Duchenne Muscular Dystrophy

Academics from Royal Holloway, University of London and a team lead by scientists at the UCL Institute of Child Health (ICH), have made an important breakthrough in the development of a treatment for Duchenne Muscular Dystrophy (DMD) which in the future could help the likes of the Lloyd family who were featured on last night's X-Factor show.

29 Nov 2011

Researchers to begin drug development projects for rare and neglected diseases

Researchers will begin drug development projects for rare and neglected diseases that include potential treatments for a musculoskeletal disorder, a cognitive dysfunction disorder, a virus that affects the central nervous system of newborns, a parasitic worm infection, a form of muscular dystrophy and a rare lung disease.

16 Nov 2011

NINDS announces grant to support work of Parent Project Muscular Dystrophy

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 that it has received a grant from the National Institutes of Neurological Disorders and Stroke (NINDS), part of the National Institutes for Health.

14 Oct 2011

Scientisits uncover evidence of powerful new cancer genetic networks

In findings with major implications for the genetics of cancer and human health, researchers at Beth Israel Deaconess Medical Center (BIDMC) and two other science teams in New York City and Rome have uncovered evidence of powerful new genetic networks and showed how it may work to drive cancer and normal development.

14 Oct 2011

Communication breakdown between nerves and muscles in SMA mouse model

Researchers at the University of Missouri have identified a communication breakdown between nerves and muscles in mice that may provide new insight into the debilitating and fatal human disease known as spinal muscular atrophy.

27 Sep 2011

AMT, NIH enter exclusive license agreement for use of AAV5-based gene therapy vectors

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has entered into an exclusive license agreement with the US National Institutes of Health (NIH) for use of adeno-associated virus serotype 5 (AAV5)-based gene therapy vectors for liver and brain indications.

6 Sep 2011

AMT total net loss decreases 7% to € 8.7 million for first half year of 2011

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the first half year of 2011.

25 Aug 2011

MDA announces $13.7M in grants to 40 new muscular dystrophy research initiatives

The Muscular Dystrophy Association today announced funding, totaling $13.7 million, for 40 new research initiatives targeting nearly two dozen progressive neuromuscular diseases.

24 Aug 2011

AVI BioPharma, MDA to initiate exon-51 Phase 2 trial in Duchenne muscular dystrophy

More than a decade of targeted Muscular Dystrophy Association-funded research, made possible as a result of generous public support of the MDA Labor Day Telethon and thousands of grass-roots special events, has today culminated in MDA providing financial assistance for the start of the first Phase 2 placebo-controlled, multiple dose efficacy, safety, tolerability and pharmacokinetics clinical trial of an exon-51 skipping drug, eteplirsen, as a potential therapy for Duchenne muscular dystrophy.

16 Aug 2011

Researchers discover biological mechanism behind sarcopenia

Researchers at Columbia University Medical Center have discovered the biological mechanism behind age-related loss of muscle strength and identified a drug that may help reverse this process.

3 Aug 2011

Positive results from phase Ib/IIa trial of novel molecular technique for Duchenne muscular dystrophy

A molecular technique originally developed at the University of North Carolina at Chapel Hill has taken one step closer to becoming a treatment for the devastating genetic disease Duchenne muscular dystrophy.

27 Jul 2011

Promising drug for Duchenne muscular dystrophy

Duchenne muscular dystrophy is a severely debilitating and lethal condition that affects 1 in 3,000. Females and males are affected, though females are rarely affected and are more often carriers. It is caused due to a defective gene that normally codes for the protein dystrophin, which helps provide a protective membrane around muscle fibers. Without this skin, muscle fibers become damaged and eventually die.

25 Jul 2011

Small molecule treatment holds promise for Usher syndrome

New treatment approach shall soon be ready for use in Usher syndrome patients / Publication in "Human Gene Therapy".

1 Jul 2011

Glybera gene therapy reduces pancreatitis risk in Lipoprotein Lipase Deficiency patients

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today released data showing that its gene therapy Glybera® (alipogene tiparvovec) significantly reduces the risk of pancreatitis in patients with Lipoprotein Lipase Deficiency (LPLD). Pancreatitis, or inflammation of the pancreas, is a major clinical symptom of LPLD.

29 Jun 2011

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera as a potential therapy for Lipoprotein Lipase Deficiency.

24 Jun 2011

Tweaking gene in satellite cells may increase lifespan of people with muscular dystrophy

Researchers have long questioned why patients with Duchenne muscular dystrophy (DMD) tend to manage well through childhood and adolescence, yet succumb to their disease in early adulthood, or why elderly people who lose muscle strength following bed rest find it difficult or impossible to regain.

18 Apr 2011

Cedars-Sinai receives $750,000 grant from Parent Project Muscular Dystrophy for sildenafil/taladafil study

Parent Project Muscular Dystrophy (PPMD) – the largest, most comprehensive non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne) – announced today that they will award Ronald G. Victor, M.D. of Cedars-Sinai Medical Center, a $750,000 grant to fund his study of phosphodiesterase inhibitors (sildenafil and taladafil) as a possible therapy for Duchenne.

5 Apr 2011

Cellectis 2010 total operating revenues increase 55% to EUR 15.8M

Cellectis, the genome engineering specialist, has announced its consolidated financial results for the financial year ended December 31, 2010.

31 Mar 2011

New insight into Duchenne muscular dystrophy

Armed with a zebrafish model of Duchenne muscular dystrophy and a library of 1,200 chemicals already approved for human use, researchers at Children's Hospital Boston have identified a compound that reverses the loss of muscle structure and function associated with DMD, seemingly by compensating for the loss of a critical protein.

19 Mar 2011