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.

Phase 3 trial of ataluren commences

PTC Therapeutics, Inc. (PTC) today announced the initiation of a Phase 3 trial of ataluren (formerly PTC124(R)), an investigational protein restoration therapy in patients with nonsense mutation cystic fibrosis (nmCF).

11 Sep 2009

Acceleron's Phase 1 trial of ACE-031 reveals its potential in treating neuromuscular diseases

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including red blood cells, bone, and muscle, today announced preliminary results from the ACE-031 Phase 1 single dose clinical trial demonstrating that ACE-031 increased lean body mass and muscle volume. The results from this randomized, placebo-controlled study were presented at the 14th International Congress of the World Muscle Society in Geneva, Switzerland.

10 Sep 2009

Human neural stem cells to treat Pelizaeus-Merzbacher Disease

StemCells, Inc. (NASDAQ:STEM) announced today that it will soon initiate with the University of California, San Francisco (UCSF) Children’s Hospital a Phase I clinical trial to evaluate the therapeutic potential of StemCells’ proprietary HuCNS-SC® product candidate (purified human neural stem cells) to treat Pelizaeus-Merzbacher Disease (PMD), a myelination disorder that primarily affects infants and young children.

10 Sep 2009

Safeway raises funds to support neuromuscular disease medical research and therapies

Safeway Inc. announced today it has raised a record $10.2 million to support medical research and services for the millions of people living with neuromuscular diseases. The funds were raised during Safeway’s annual Muscular Dystrophy Association (MDA) campaign, a five-week endeavor to highlight the need to find cures and treatments for muscular dystrophy and related diseases, and presented during the Jerry Lewis MDA Telethon in Las Vegas Labor Day weekend.

9 Sep 2009

Small molecule blocks aberrant pathway associated with myotonic dystrophy

Researchers at the University of Illinois have designed a small molecule that blocks an aberrant pathway associated with myotonic dystrophy type 1, the most common form of muscular dystrophy.

8 Sep 2009

Muscular Dystrophy Canada to host the 44th Jerry Lewis Labour Day Telethon

Muscular Dystrophy Canada is hosting the 44th Annual Jerry Lewis Labour Day Telethon to support those living with a neuromuscular disorder.

7 Sep 2009

Victim’s longevity with the deadly disease inspires thousands of dying patients

When Jerry Lewis takes the stage again on Labor Day 2009 for the Muscular Dystrophy Association Telethon, thousands of people who suffer from the same lung disease as he will be watching - and not just to hear the funny things he'll say. They'll be looking for inspiration from the funny man who suffers from a not-so-funny and little known fatal lung disease.

5 Sep 2009

Children International distributes free wheelchairs to physically disabled children in Ecuador

For 10-year-old Jessica, who struggles with muscular dystrophy and epilepsy that make her life on the steep mountainside above Quito, Ecuador extremely difficult, her new wheelchair is priceless!

4 Sep 2009

Celgene to collaborate with PTC on advancing drug discovery efforts

PTC Therapeutics, Inc. (PTC) today announced that Celgene Corporation has exercised its option to collaborate on advancing drug discovery efforts on an oncology target addressed through the application of PTC's GEMS(TM) (Gene Expression Modulation by Small-molecules) technology.

3 Sep 2009

Phase 1 single dose clinical trial of Acceleron Pharma's ACE-031 increases lean body mass

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including red blood cells, bone, and muscle, today announced preliminary results from the ACE-031 Phase 1 single dose clinical trial indicating that ACE-031 was safe and well-tolerated at all dose levels and increased lean body mass.

2 Sep 2009

Neuromuscular disorders common among Canadians

September is Muscular Dystrophy Awareness Month. There are more than 100 neuromuscular disorders and it is estimated more than 50,000 Canadians are affected by one form or another. Each form is caused by an error in a specific gene related to muscle function.

2 Sep 2009

AVI BioPharma's peptide-conjugated PMO chemistry to be discussed at two upcoming scientific meetings

AVI BioPharma, Inc., a developer of RNA-based drugs, today announced that Hong Moulton, Ph.D., Director of Discovery Research, will give oral presentations highlighting improved analogues of AVI's phosphorodiamidate morpholino oligomer (PMO) chemistry at two upcoming scientific meetings.

28 Aug 2009

ClubCorp partners with Muscular Dystrophy Association to raise funds to support ALS research initiative and more than 100 nonprofit groups

ClubCorp, The World Leader in Private Clubs, announced today that it is hosting the third annual ClubCorp Charity Classic in partnership with the Muscular Dystrophy Association (MDA). This event is the world's largest multi-site charity golf and dining event, with participating clubs teeing up across the country from California to New York.

26 Aug 2009

Amsterdam Molecular Therapeutics reports 2009 half year financials

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today reported its results for the first half year of 2009.

13 Aug 2009

New approach to remove toxic agent that causes myotonic dystrophy

Cell biologists from the Radboud University Nijmegen Medical Centre (Nijmegen, the Netherlands) describe a new approach to remove the toxic agent that causes the neuromuscular disease myotonic dystrophy. Their findings are published in the scientific journal The Proceedings of the National Academy of Sciences.

11 Aug 2009

Chemists rationally design inhibitors against an RNA molecule that causes myotonic muscular dystrophy

Chemists at the University at Buffalo have used rational drug design to synthesize small, cell-permeable molecules that are effective in vitro against two common types of myotonic muscular dystrophy, a result that has implications for potentially curing muscular dystrophy, as well as other diseases.

9 Aug 2009

New function for missing protein in Duchenne muscular dystrophy found

Researchers at the University of Minnesota and National Institutes of Health have identified a new function for the protein missing in people with the most common and ultimately lethal form of childhood muscular dystrophy.

3 Aug 2009

Sticky protein protects cells from mechanical stress

A new study by scientists at the University of Iowa shows why muscle membranes don't rupture when healthy people exercise.

26 Jul 2009

Genetic source of muscular dystrophy blocked

Researchers at the University of Rochester Medical Center have found a way to block the genetic flaw at the heart of a common form of muscular dystrophy. The results of the study, which were published today in the journal Science, could pave the way for new therapies that essentially reverse the symptoms of the disease.

17 Jul 2009

New drug for muscle degenerating diseases shows great promise

A new drug being studied for the treatment of muscle degenerating diseases has shown promising results. According to a study published today in the British Journal of Pharmacology, Debio 025 is as effective as current drugs but, crucially, does not cause unwanted immunosuppressive effects.

10 Jun 2009