Neuromuscular Disease News and Research

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Women riders support MDA's initiative on neuromuscular disease

During this year's 69th Annual Daytona Bike Week, hundreds of female motorcycle enthusiasts and Harley-Davidson Motor Company are banding together to support the Muscular Dystrophy Association (MDA) and their efforts to find a cure for neuromuscular disease.

6 Feb 2010

PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently.

20 Jan 2010

microRNA-206 plays crucial role in the progression of ALS

miRagen Therapeutics, Inc., a biopharmaceutical company focused on improving patients' lives by developing innovative microRNA-based therapeutics for cardiovascular and muscle disease, today announced the publication of data demonstrating that microRNA-206 (miR-206) plays a crucial role in the progression of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) and in neuromuscular synaptic regeneration in mice.

11 Dec 2009

Non-clinical data of CK-2017357 presented by Cytokinetics

Cytokinetics, Incorporated announced today that both a poster and an oral presentation summarizing non-clinical data regarding CK-2017357, a novel fast skeletal troponin activator, were presented at the 5th Cachexia Conference, organized by the Society on Cachexia and Wasting Disorders, being held December 5-8, 2009 in Barcelona, Spain.

8 Dec 2009

Myasthenia Gravis Alliance receives Global Pharmaceuticals grant to further research and awareness

Global Pharmaceuticals, the generic products division of Impax Laboratories, Inc., today announced their cooperation in awarding a modest grant to Myasthenia Gravis Alliance, also known as Myasthenia Gravis Foundation of Greater New York, Inc., a 501(3)(c) non profit membership corporation, incorporated in the State of New York.

25 Nov 2009

UT physicians treat Iraqi girl with myasthenia gravis

Plagued by a growing weakness that left her unable to walk, talk and even take a normal breath, 11-year-old Iraqi Aram Ali was a shell of the bright little girl she used to be.

16 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

Acceleron Pharma commences Phase 1 clinical study of its novel angiogenesis inhibitor

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including red blood cells, bone, muscle, fat, and the vasculature, today announced the initiation of a Phase 1 clinical study of ACE-041, a novel angiogenesis inhibitor that binds to and prevents members of the TGF-β superfamily from signaling through the activin receptor-like kinase 1 (ALK1) receptor.

4 Nov 2009

CIRM awards Salk Institute $10.8M grant to develop stem-cell based therapy for Lou Gehrig's Disease

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.

29 Oct 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

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

Cytrx to submit revised protocol for arimoclomol Phase IIb clinical trial

CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company engaged in the development of high-value human therapeutics, today announced its intention during the 2009 fourth quarter to submit a revised protocol for its planned Phase IIb clinical trial for arimoclomol as a treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The decision follows receipt of an anticipated written letter from the U.S.

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

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

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

Electrodiagnostic and neuromuscular medicine featured at AANEM annual meeting

The American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) will hold its 56th annual meeting in San Diego, California, from October 7-10, 2009 at the Manchester Grand Hyatt.

10 Aug 2009

Nervous system may be culprit in Pompe disease

Brain may win out over brawn as the primary cause of breathing problems in children with a severe form of muscular dystrophy known as Pompe disease.

25 May 2009

Exercise therapy benefits ICU patients

An exercise program developed at Wake Forest University Baptist Medical Center aimed at helping patients in the medical Intensive Care Unit (ICU) maintain physical conditioning is a featured 'Innovation Profile' on the Agency for Healthcare Research and Quality (AHRQ) Web site.

23 Mar 2009

Carrier screening for spinal muscular atrophy should be offered to all couples

Carrier screening for spinal muscular atrophy (SMA) - a serious genetic disease affecting approximately 1 in 10,000 infants that causes progressive muscle weakness and death - should be made available to all families, according to a new practice guideline issued by the American College of Medical Genetics (ACMG).

10 Dec 2008