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.

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

Researchers find compound that reverses type 1 myotonic dystrophy

A compound already used to treat pneumonia could become a new therapy for an inherited muscular wasting disease, according to researchers at the University of Oregon and the University of Rochester School of Medicine and Dentistry in New York.

7 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

Phase 2a clinical trial in nonsense mutation hemophilia A and hemophilia B initiated

PTC Therapeutics, Inc. (PTC) today announced it is expanding the development of ataluren, an investigational new drug, to a third indication with the initiation of a Phase 2a clinical trial in nonsense mutation hemophilia A (nmHA) and hemophilia B (nmHB).

26 Oct 2009

H.R. 3790 wins praises in Pennsylvania

The Pennsylvania Association of Medical Suppliers and the American Association for Homecare praised a bipartisan bill in Congress, H.R. 3790, to eliminate the misguided and deeply flawed "competitive" bidding program for durable medical equipment and services in Medicare. The bidding process in this controversial program began on October 21 in the Pittsburgh metropolitan statistical areas and seven other MSAs across the U.S.

23 Oct 2009

Bill eliminating the flawed medical equipment bidding process in the Riverside-San Bernardino area lauded

The California Association of Medical Product Suppliers and the American Association for Homecare praised a bipartisan bill in Congress, H.R. 3790, to eliminate the misguided and deeply flawed "competitive" bidding program for durable medical equipment and services in Medicare.

23 Oct 2009

Texas and U.S. homecare groups praise bipartisan bill; say bidding program may reduce quality patient care

The Texas Alliance for Home Care Services and the American Association for Homecare praised a bipartisan bill in Congress, H.R. 3790, to eliminate the deeply flawed "competitive" bidding program for durable medical equipment and services in Medicare.

23 Oct 2009

H.R. 3790 to reduce Medicare spending; preserve access to quality care

The Ohio Association of Medical Equipment Services and the American Association for Homecare praised a bipartisan bill in Congress, H.R. 3790, to eliminate the misguided, flawed "competitive" bidding program for durable medical equipment and services in Medicare.

22 Oct 2009

American Association for Homecare commends bipartisan lawmakers

The American Association for Homecare praised a bipartisan group of lawmakers in the House of Representatives for backing introduction of H.R. 3790, a bill to eliminate the controversial, deeply flawed "competitive" bidding program for durable medical equipment and services in Medicare.

21 Oct 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

Older generation antibiotic for treating colon cancer

A new Tel Aviv University drug, based on an older generation antibiotic, may provide doctors with an effective and innovative method of treating colon cancer in both its incipient and full-blown stages ― and minimize the need for painful, uncomfortable colonoscopies and surgical polyp removal.

14 Oct 2009

Mouse model of ALS closely resembles humans with the paralyzing disorder, says report

A new mouse model of amyotrophic lateral sclerosis (ALS) closely resembles humans with the paralyzing disorder, researchers at Washington University School of Medicine in St. Louis report.

13 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

HDSA honors outstanding contributors against Huntington's Disease

The Huntington's Disease Society of America (HDSA), will recognize the outstanding contributions to improving the quality of life of people with Huntington's Disease (HD) by several individuals and two organizations at the 13th annual Guthrie Awards Dinner this Thursday.

7 Oct 2009

Research to explore diagnosis and treatment of rare diseases

The National Institutes of Health announced today a second phase of the Rare Diseases Clinical Research Network (RDCRN) including funds for 19 research consortia.

7 Oct 2009

UCLA discovery raises hope for curing genetic disorders caused by nonsense mutations

UCLA scientists have identified two chemicals that convince cells to ignore premature signals to stop producing important proteins. Published in the Sept. 28 edition of the Journal of Experimental Medicine, the findings could lead to new medications for genetic diseases, such as cancer and muscular dystrophy, that are sparked by missing proteins.

29 Sep 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

'Pathway to a Cure' walk event in California

Satellite Healthcare, one of the nation's first and leading providers of kidney dialysis services and a major sponsor of nephrology research, today announced their sponsorship of The Polycystic Kidney Disease Foundation's "Pathway to a Cure" walk event in California's capital of Sacramento.

18 Sep 2009

NIH to fund grants for epigenetic research

The National Institutes of Health announced today that it will fund 22 grants on genome-wide studies of how epigenetic changes -- chemical modifications to genes that result from diet, aging, stress, or environmental exposures -- define and contribute to specific human diseases and biological processes.

17 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

Muscular Dystrophy News and Research

Further Reading

AMT's gene therapy successful in treating Duchenne muscular dystrophy

Researchers find compound that reverses type 1 myotonic dystrophy

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

Phase 2a clinical trial in nonsense mutation hemophilia A and hemophilia B initiated

H.R. 3790 wins praises in Pennsylvania

Bill eliminating the flawed medical equipment bidding process in the Riverside-San Bernardino area lauded

Texas and U.S. homecare groups praise bipartisan bill; say bidding program may reduce quality patient care

H.R. 3790 to reduce Medicare spending; preserve access to quality care

American Association for Homecare commends bipartisan lawmakers

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

Older generation antibiotic for treating colon cancer

Mouse model of ALS closely resembles humans with the paralyzing disorder, says report

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

HDSA honors outstanding contributors against Huntington's Disease

Research to explore diagnosis and treatment of rare diseases

UCLA discovery raises hope for curing genetic disorders caused by nonsense mutations

Macrophages play a crucial role in muscle regeneration

'Pathway to a Cure' walk event in California

NIH to fund grants for epigenetic research

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

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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