Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

University of Antioquia and IBM partner for Leishmaniasis research

IBM and University of Antioquia's Tropical Disease Study and Control Program (PECET) today announced a joint research project, Drug Search for Leishmaniasis, using computer power from IBM's World Community Grid.

14 Sep 2011

New treatment strategy for dominant forms of muscular dystrophy

Investigators at The Research Institute at Nationwide Children's Hospital are studying a potential new treatment strategy for dominant forms of muscular dystrophy, thanks to preliminary funding from The Ohio State University Center for Clinical and Translational Science.

12 Sep 2011

Pharnext puts together €2.5M to fund development of Pleodrug for Charcot-Marie-Tooth disease

Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments based on Pleotherapy for severe neurological diseases, today announced that it has put together a €2.5 million round of financing (corresponding to a pre-money company valuation of €60 million).

8 Sep 2011

Study on effects of adult stem cell treatments in patients with Hereditary Ataxia

The Journal of Translational Medicine reports on a new study in which 30 patients with Hereditary Ataxia showed statistically significant improvements in functionality and quality of life after being treated with a combination of cord blood-derived stem cell treatments and physical therapy.

7 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

PTC restructures collaboration with Genzyme

PTC Therapeutics, Inc. and Genzyme, a Sanofi company, announced today the restructuring of their collaboration. Under the original agreement, PTC held commercial rights for the U.S. and Canada and Genzyme held commercial rights in all other countries.

2 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

Consumer groups, economists and members of Congress oppose Medicare's bidding system

As Medicare expands a controversial "competitive" bidding program for home medical equipment and services, economists, consumer groups, and members of Congress have gone on record to oppose that program citing reduced patient access to care, flaws in the program design, and impact on local jobs.

20 Aug 2011

Gene therapy cocktail can treat inherited diseases with 'misfolded' proteins

In a kind of molecular gymnastics, scientists at the University of North Carolina at Chapel Hill School of Medicine have devised a gene therapy cocktail that has the potential to treat some inherited diseases associated with "misfolded" proteins.

16 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

Noninvasive brain computer interface could turn thoughts into motion

"Brain cap" technology being developed at the University of Maryland allows users to turn their thoughts into motion. Associate Professor of Kinesiology José 'Pepe' L. Contreras-Vidal and his team have created a non-invasive, sensor-lined cap with neural interface software that soon could be used to control computers, robotic prosthetic limbs, motorized wheelchairs and even digital avatars.

28 Jul 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

FDA approves U.S. launch of Wiskott-Aldrich gene therapy clinical trial

The U.S. Food & Drug Administration (FDA) approved the launching in the U.S. of a clinical trial for gene therapy for a rare immunodeficiency, Wiskott-Aldrich syndrome.

22 Jul 2011

Researchers identify gene linked to muscle endurance

Researchers at the Perelman School of Medicine at the University of Pennsylvania have identified a gene for endurance, or more precisely, a negative regulator of it.

19 Jul 2011

Researchers discover crucial role of miR-206 in skeletal muscle development

Research led by the University of East Anglia has discovered the crucial role of a molecule in skeletal muscle development.

5 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