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Muscular Dystrophy News
Smoking May Now Be Considered An Established Risk Factor For ALS
While previous studies have indicated a "probable" connection between smoking and ALS, a new study published in the Nov. 17, 2009 issue of Neurology®, the medical journal of the American Academy of Neurology, states that smoking may now be considered an "established" risk factor for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. The findings come from Baystate Medical Center neurologist Dr. ...more
18 Nov 2009
Scientists Follow Their Noses When Investigating Muscle Repair
When muscle cells need repair, they use odor-detecting tools found in the nose to start the process, researchers have discovered. The results are published online and scheduled for publication in the November issue of the journal Developmental Cell Found on the surfaces of neurons inside the nose, odorant receptors are molecules that bind and respond to substances wafting through the air. ...more
17 Nov 2009
Statement On The Baby RB Case - Muscular Dystrophy Campaign
A baby with a rare neuromuscular condition who has been on a ventilator in hospital since birth, known for legal reasons as Baby RB, has been at the centre of a legal debate between his parents - a debate which has now ended with the baby's father withdrawing his objections to the ventilator being switched off. He was born with congenital myasthenic syndrome, a muscle weakness that limits the movement of his limbs and his ability to breathe on his own. ...more
15 Nov 2009
Tesco Takes The Biscuit For Charity - Muscular Dystrophy Campaign, UK
Tesco customers are being asked to get dunking to raise at least £1,500 for children with muscle-wasting diseases. The retailer has created this limited edition biscuit exclusively to raise funds for the Muscular Dystrophy Campaign, Tesco Charity of the Year 2009. ...more
15 Nov 2009
South Florida Man Inspires 'Ian's Law'
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. ...more
12 Nov 2009
Treatment To Improve Degenerating Muscle Gains Strength
A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders. Severe weakness of the quadriceps is a defining feature of several neuromuscular disorders. ...more
12 Nov 2009
MDA Grantees Prove Gene Therapy Grows Muscle Strength & Size In Primates
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. 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
12 Nov 2009
North East MP Takes Fight For Muscle Disease Patients To The Commons
MP Dave Anderson fought for better services for children and adults with neuromuscular conditions during a House of Commons debate when he called for "real improvements throughout the country" for patients earlier this week. Mr Anderson, the Chair of the All Party Parliamentary Group for Muscular Dystrophy also met with ministers and peers to look forward to the launch of a new investigation and subsequent report into wheelchair provision for people with muscle disease. ...more
10 Nov 2009
Possible Help In Fight Against Muscle-wasting Disease
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
10 Nov 2009
PUrsuing Novel Stem-Cell Derived Therapy For Lou Gehrig's Disease With $10.8 Million Award
The Salk Institute has been awarded a $10.8 million grant by the California Institute for Regenerative Medicine (CIRM) for translational research focusing on developing a novel stem-cell based therapy for Amyotrophic Lateral Sclerosis (ALS) - or Lou Gehrig's Disease. Sam Pfaff, Ph.D. ...more
30 Oct 2009
AVI BioPharma Presents Updated Safety Data From Ongoing Systemic Trial Of AVI-4658 At 7th Annual Action Duchenne International Conference
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, presented an update on preliminary safety data from its ongoing systemic Phase 1b/2 clinical trial of exon skipping AVI-4658 in patients with Duchenne muscular dystrophy (DMD) at the 7th Annual Action Duchenne Conference in London, UK. ...more
28 Oct 2009
$3.7 Million NIH Grant Will Fund Study On Stem Cells Derived From ALS Patients
Johns Hopkins scientists have been awarded a $3.7 million grant from the National Institutes of Health (NIH) to learn more about the nerve and muscle-wasting disease amyotrophic lateral sclerosis (ALS) using stem cells developed from ALS patients' skin. The award, given over a two-year span, will be shared with three other laboratories, including one at Harvard University and two at Columbia University. The Johns Hopkins team, led by neurologist Jeffrey Rothstein, M.D., Ph.D. ...more
23 Oct 2009
AVI BioPharma To Present Safety Update From Ongoing Systemic Trial Of AVI-4658 At 7th Annual Action Duchenne International Conference
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, announced that it will present updated preliminary safety data from its ongoing systemic Phase 1b/2 clinical trial of AVI-4658 in patients with Duchenne muscular dystrophy (DMD) at the 7th Annual Action Duchenne Conference taking place Oct. 23-24 in London, UK. Steve Shrewsbury, M.D. ...more
23 Oct 2009
A Promising New Development For Treatment Of Duchenne Muscular Dystrophy
A technique called exon skipping shows great potential to increase muscle strength and prolong life in people with a severe form of Duchenne muscular dystrophy (DMD). According to a study that included MDA-supported Stephen Wilton at the University of Western Australia, exon skipping improves production of a crucial muscle protein that's missing in people with DMD. For the first time, these results were observed in mice with an especially severe form of muscular dystrophy. ...more
23 Oct 2009
Exon Skipping Drug Prevents Muscle Wasting And Maintains Muscle Function In Severely Affected, Dystrophin Deficient Mice
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. These findings were published online today in the journal Molecular Therapy and support the promise of this therapeutic approach for the treatment of Duchenne muscular dystrophy (DMD). ...more
22 Oct 2009
Compound Shows Potential For Slowing Progression Of ALS
A chemical cousin of a drug currently used to treat sepsis dramatically slows the progression of amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig's disease, in mice. The results offer a bit of good news in efforts to develop a therapy to stop or slow the progression of a disease that generally kills its victims within just a few years. In a paper published online Oct. ...more
20 Oct 2009
Dysfunctional Protein Dynamics Behind Neurological Disease?
Researchers at Lund University, Sweden, have taken a snapshot of proteins changing shape, sticking together and creating structures that are believed to trigger deadly processes in the nervous system. The discovery opens the possibility of designing drugs for a devastating neurological disease, ALS. ...more
14 Oct 2009
LSUHSC's Dr. John England Plays Key Role In Developing New ALS Treatment Guidelines
Dr. John England, Professor and Chairman of Neurology at LSU Health Sciences Center New Orleans, analyzed research findings and was responsible for the quality and accuracy of evidence analysis and the conclusions of the studies resulting in new guidelines for treating Lou Gehrig's disease, or amyotropic lateral sclerosis (ALS). The guidelines will be published in the October 13, 2009 issue of Neurology, the journal of the American Academy of Neurology. ...more
13 Oct 2009
Best Treatments To Help ALS Patients Live Longer, Easier: New Guidelines
New guidelines from the American Academy of Neurology identify the most effective treatments for amyotrophic lateral sclerosis (ALS), often called Lou Gehrig's disease. The guidelines are published in the October 13, 2009, issue of Neurology®, the medical journal of the American Academy of Neurology. "While we are waiting for a cure, people need to know that a lot can be done to make life easier and longer for people with ALS," said lead guidelines author Robert G. ...more
13 Oct 2009
Muscular Dystrophy Association Applauds New Federal Support For Neuromuscular Diseases
The National Institutes of Health has announced a $117 million expansion of its Rare Diseases Clinical Research Network, adding 14 research consortia, or groups, to the Network's second phase and will continue funding the five original groups. Having long recognized the research challenges for communities affected by rare disorders, the National Institutes of Health established the Office of Rare Disease Research in 1993 to ensure an increased focus on rare disorder treatment discovery. ...more
10 Oct 2009

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