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.

Scientists identify gene underlying a disease that causes temporary paralysis of skeletal muscle

Scientists have identified a gene underlying a disease that causes temporary paralysis of skeletal muscle. The finding, they say, illustrates how investigations of rare genetic diseases can drive insights into more common ones.

8 Jan 2010

Muscular Dystrophy Association announces milestone grant to develop new treatments for ALS

Buoyed by the extraordinary progress being made by the ALS Therapy Development Institute, the Muscular Dystrophy Association today announced a new milestone-driven grant of $2.5 million, adding to the $18 million MDA already has invested with ALS TDI -- the world's only non-profit research center focused exclusively on developing treatments for amyotrophic lateral sclerosis (ALS).

7 Jan 2010

Findings open up significant possibilities for developing micro-screening devices for drug discovery

A Nobel-winning process for testing new drugs to treat diseases such as Huntington's, Parkinson's, and muscular dystrophy is getting an electrical charge.

6 Jan 2010

AMT receives Innovation Credit to develop gene therapy treatment for DMD

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that it will receive an Innovation Credit of up to EUR 4 million from the Dutch government to support the development of AMT's gene therapy treatment for Duchenne Muscular Dystrophy (DMD).

6 Jan 2010

UI cell membrane protein study may lead to new methods of tracking metastasizing cancer cells

With the discovery of a new type of chemical modification on an important muscle protein, a University of Iowa study improves understanding of certain muscular dystrophies and could potentially lead to new treatments for the conditions.

4 Jan 2010

Tarantula venom protein promising for MD

University at Buffalo biophysicists have found a protein in tarantula venom that shows promise as a potential therapy for muscular dystrophy (MD). They have formed a start-up biotech company in Buffalo -- Rose Pharmaceuticals -- to advance the drug to clinical trials.

30 Dec 2009

Repligen receives $731,000 to support ongoing development of new treatments for Friedreich's ataxia

Repligen Corporation today announced that the Company has received $731,000 in research funding to support the ongoing development of new treatments for Friedreich's ataxia from the Muscular Dystrophy Association (MDA).

16 Dec 2009

ALS TDI and Applied Proteomics join to identify protein biomarkers associated with ALS

The ALS Therapy Development Institute (ALS TDI) and Applied Proteomics, Inc. announced today that they have completed the first stage of a multi-year collaboration to identify and validate protein biomarkers associated with amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease).

15 Dec 2009

Kennedy Krieger Institute opens new center for individuals with muscle disorders

Kennedy Krieger Institute announced today the opening of the new Center for Genetic Muscle Disorders that will improve and expand services to individuals with muscle disorders such as muscular dystrophy and congenital myopathy in the Baltimore/Washington region and across the nation. The Center provides expert clinical care to children and adults with these disorders, and offers leading research programs both in the clinical and laboratory setting.

9 Dec 2009

Humans and mice have critical differences in gene responsible for DMD

Humans and mice have previously unknown and potentially critical differences in one of the genes responsible for Duchenne muscular dystrophy (DMD). Researchers writing in the open access journal BMC Biology have found that two major features of a key DMD gene are present in most mammals, including humans, but are specifically absent in mice and rats, calling into question the use of the mouse as the principal model animal for studying DMD.

4 Dec 2009

ISIS-FXIRx and ISIS-SMNRx included in Isis Pharmaceuticals' development pipeline

Isis Pharmaceuticals, Inc. announced today that it has added two new drugs to its development pipeline, ISIS-FXIRx and ISIS-SMNRx.

4 Dec 2009

Combination of green tea and DAPH-12 chemicals may help prevent brain disorders

Scientists at Boston Biomedical Research Institute (BBRI) and the University of Pennsylvania have found that combining two chemicals, one of which is the green tea component EGCG, can prevent and destroy a variety of protein structures known as amyloids. Amyloids are the primary culprits in fatal brain disorders such as Alzheimer's, Huntington's, and Parkinson's diseases.

4 Dec 2009

Discovery of cell fibrous structure may provide clues to diagnose and treat medical disorders

Researchers from the Johns Hopkins Engineering in Oncology Center have shown that in healthy cells, a bundled "cap" of thread-like fibers holds the cell's nucleus, its genetic storehouse, in its proper place.

3 Dec 2009

StemCells commences patient recruitment for a Phase I clinical trial of HuCNS-SC in PMD

StemCells, Inc. announced today that it has commenced patient recruitment for a Phase I clinical trial designed to test the safety and preliminary efficacy of its HuCNS-SC® purified human neural stem cells in Pelizaeus-Merzbacher Disease (PMD), a neurological disorder that primarily afflicts children. The study is being conducted at the University of California, San Francisco (UCSF) Children’s Hospital, one of the leading medical centers in the United States for neonatology, pediatric neurology and neurosurgery.

24 Nov 2009

Amsterdam Molecular Therapeutics provides third-quarter 2009 business updates

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.

18 Nov 2009

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

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.

17 Nov 2009

Galapagos third quarter 2009 financial results in line with management expectations

Galapagos NV announces today that third quarter 2009 operational and financial results were in line with management expectations. These results encourage the Company to retain full year guidance of EUR 100 million revenues, a growth of 30% compared to 2008. Galapagos' service division BioFocus will significantly increase its cash contribution to the Group result for the full year 2009.

13 Nov 2009

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

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.

12 Nov 2009

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

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

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."

12 Nov 2009

Muscular Dystrophy News and Research

Further Reading

Scientists identify gene underlying a disease that causes temporary paralysis of skeletal muscle

Muscular Dystrophy Association announces milestone grant to develop new treatments for ALS

Findings open up significant possibilities for developing micro-screening devices for drug discovery

AMT receives Innovation Credit to develop gene therapy treatment for DMD

UI cell membrane protein study may lead to new methods of tracking metastasizing cancer cells

Tarantula venom protein promising for MD

Repligen receives $731,000 to support ongoing development of new treatments for Friedreich's ataxia

ALS TDI and Applied Proteomics join to identify protein biomarkers associated with ALS

Kennedy Krieger Institute opens new center for individuals with muscle disorders

Humans and mice have critical differences in gene responsible for DMD

ISIS-FXIRx and ISIS-SMNRx included in Isis Pharmaceuticals' development pipeline

Combination of green tea and DAPH-12 chemicals may help prevent brain disorders

Discovery of cell fibrous structure may provide clues to diagnose and treat medical disorders

StemCells commences patient recruitment for a Phase I clinical trial of HuCNS-SC in PMD

Amsterdam Molecular Therapeutics provides third-quarter 2009 business updates

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

Galapagos third quarter 2009 financial results in line with management expectations

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

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

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

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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