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.

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

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

Muscular Dystrophy News and Research

Further Reading

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

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

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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