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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.

Amsterdam Molecular Therapeutics provides third-quarter 2009 business updates

18. November 2009 04:03
Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today provides its non-audited business update in compliance with the EU transparency directive. This report summarizes material events and AMT's financial position for the third quarter of 2009. [More]

Pet therapy can have a positive effect on patients’ physical and psychosocial well being

17. November 2009 00:17
Adults who use pet therapy while recovering from total joint-replacement surgery require 50 percent less pain medication than those who do not. These findings were presented at the 18th Annual Conference of the International Society of Anthrozoology and the First Human Animal Interaction Conference (HAI) in Kansas City, Mo. [More]

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Gene therapy increases muscle size and strength with no adverse effects in monkeys: MDA

12. November 2009 04:21
Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength and no adverse effects, the Muscular Dystrophy Association (MDA) announced today. The findings could have implications particularly for injured and aging people worldwide; and for tens of millions experiencing muscle loss associated with cancer, AIDs and muscle diseases. [More]

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

12. November 2009 00:50
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. [More]

South Florida man inspires "Ian's Law," fights against discriminatory health insurance practices

12. November 2009 00:41
McClatchy/The Miami Herald reports on Ian Pearl, a South Florida man born with muscular dystrophy who is drawing attention to discriminatory health insurance practices against the disabled. Pearl is the "inspiration for 'Ian's Law,' legislation being introduced by two New York state legislators that would require insurance companies to get approval from the state before dropping coverage plans for existing clients." [More]

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AMT's gene therapy successful in treating Duchenne muscular dystrophy

11. November 2009 03:27
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. [More]

Researchers find compound that reverses type 1 myotonic dystrophy

7. November 2009 00:42
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. [More]

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

6. November 2009 23:48
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. [More]

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

26. October 2009 08:42
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). [More]

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H.R. 3790 wins praises in Pennsylvania

23. October 2009 05:00
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. [More]

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

23. October 2009 04:40
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. [More]

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

23. October 2009 03:13
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. [More]

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

22. October 2009 07:12
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. [More]

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American Association for Homecare commends bipartisan lawmakers

21. October 2009 05:43
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. [More]

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

21. October 2009 02:57
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. [More]
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