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.

NIH awards UF scientists $7.5M for study of MRI technology in evaluating children with DMD

Duchenne muscular dystrophy research at the University of Florida got a major boost with the award of $7.5 million in National Institutes of Health funding to study the use of magnetic resonance imaging in determining the natural progression of the disease.

10 May 2010

Movements of satellite cells could help patients with muscular dystrophy: MU researchers

When muscle tissue experiences trauma or disease, such as muscular dystrophy, stem cells in the muscle known as "satellite cells" respond to repair and regenerate the muscle. These cells are particularly important in neuromuscular diseases, such as muscular dystrophy, which affect muscle stability and repair.

7 May 2010

Acceleron Pharma commences Phase 2 clinical trial of ACE-031 protein therapeutic in DMD patients

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the initiation of a Phase 2 clinical trial with ACE-031 in patients with Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease. ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling of a cell surface receptor called the activin receptor type IIB (ActRIIB).

5 May 2010

Medication used to treat heart arrhythmias also reduces myotonic dystrophy: Researchers

A medication most often used to treat heart arrhythmias also reduces a central symptom of myotonic dystrophy, the most common type of muscular dystrophy in adults.

5 May 2010

BioTime acquires ES Cell International

BioTime, Inc., a biotechnology company that develops and markets products in the field of human stem cells and regenerative medicine, today announced that it has completed its acquisition of the Singapore company ES Cell International Pte Ltd.

3 May 2010

BioTime to acquire Singapore-based company, ES Cell International

BioTime, Inc., a biotechnology company that develops and markets products in the field of stem cells and regenerative medicine, today announced that it has agreed to acquire the Singapore-based company ES Cell International Pte Ltd.

30 Apr 2010

MU researcher develops test to pinpoint neurological origins of dysphagia

Muscle degeneration and confinement to a wheelchair are the hallmarks of Lou Gehrig's disease, Parkinson's, muscular dystrophy and other neurodegenerative diseases. One of the silent, and most serious, symptoms of these diseases is losing the ability to swallow.

29 Apr 2010

Pharmacological suppression of nonsense mutations effective in treating genetic defects

Genetic mutation can disrupt the way human cells make proteins, which in turn leads to inherited disease. David Bedwell, a professor in the University of Alabama at Birmingham (UAB) Department of Microbiology, says scientists are closer than ever to producing drugs that fix this disrupted-protein pathway and drastically improving treatment of genetic disease.

27 Apr 2010

Scientists now closer to produce drugs to fix disease-causing mutations, spare healthy genes

Inherited diseases such as cystic fibrosis can be caused by genetic "nonsense mutations" that disrupt the way human cells make proteins. David Bedwell, Ph.D., a professor in the University of Alabama at Birmingham Department of Microbiology, says scientists are now closer to producing drugs that will fix this disruption and drastically improve treatment of genetic disease.

27 Apr 2010

Hospital for Special Care launches ALS Clinical Trials Unit at Neuromuscular Center

Hospital for Special Care announced today the launch of the region's first Amyotrophic Lateral Sclerosis (ALS) Clinical Trials Unit at its Neuromuscular Center. The new unit will be headed by Jinsy A. Andrews, M.D., a physician researcher recruited from Columbia University. Dr. Andrews, Director of Research & Clinical Trials Unit, Hospital for Special Care, specializes in neuromuscular research and will facilitate the hospital's first clinical trial for ALS upon her arrival.

23 Apr 2010

BioMarin Pharmaceutical introduces Firdapse for LEMS in the E.U.

BioMarin Pharmaceutical Inc. announced today that Firdapse™ (3,4-diaminopyridine) is now commercially available in the European Union (E.U.) for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). Launching immediately in Germany and the UK, the company expects to subsequently launch Firdapse in all major European markets by the end of 2010.

19 Apr 2010

New therapeutic approach for Duchenne muscular dystrophy

Researchers from Universit- Laval's Faculty of Medicine and the CHUQ Research Center have proven that it is possible to repair the defective gene responsible for Duchenne muscular dystrophy.

16 Apr 2010

Embryos with two mums to remove the risk of hereditary diseases: new research

New research published in the journal Nature, could pave the way to world’s first genetically modified human baby. The research involves making embryos with genes from one man and two women. There are hundreds or families around the world who are left childless or have serious ill babies because they carry genes for hereditary illnesses. This research may bring hope to such couples. According to scientists on the team, within the next three years these women will be able to avail such techniques. There has been skepticism regarding ethical issues attached to making of “designer babies”.

16 Apr 2010

MDA awards over $21M in grants for advancing critical neuromuscular research

Forty-two medical researchers and their labs have been awarded more than $21 million in grants by the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multi-year awards to be dispersed over the next three years.

15 Apr 2010

New seminar series "Physical Care of Duchenne" unveiled

Duchenne Therapy Network today unveiled a new seminar series called, "Physical Care of Duchenne." The course, which includes two separate sessions taught by Jennifer Wallace, PT, will be held at the Ayres Hotel in Costa Mesa.

7 Apr 2010

MDA, FFSHR award $200,000 grant for developing FSH dystrophy therapy

The Muscular Dystrophy Association (MDA), headquartered in Tucson, Ariz., and Friends of FSH Research (FFSHR), based in Kirkland, Wash., has jointly awarded a two-year, $200,000 grant to Dr. Joel Chamberlain, a research assistant professor of medical genetics at the University of Washington. The grant, equally funded by the two organizations, will enable the laboratory led by Dr. Chamberlain to study RNA interference as an investigative and therapeutic tool for facioscapulohumeral muscular dystrophy.

7 Apr 2010

Barrow Neurological Institute scientist receives grant to study myasthenia gravis

A scientist at Barrow Neurological Institute in Phoenix has been awarded a major grant from the Muscular Dystrophy Association to continue his groundbreaking work in the disease of myasthenia gravis.

6 Apr 2010

Beike Biotechnology awarded ISO9001 certification for implementation of comprehensive QMS

Beike Biotechnology Co. Ltd., one of the world's leading biotechnology companies focusing on adult stem cell therapies, has been awarded ISO9001 certification by the International Organization for Standardization ("ISO"). This internationally recognized designation signifies Beike has established a Quality Management System (QMS) that complies with ISO9001 standards.

3 Apr 2010

MDA awards 42 medical researchers and labs over $21M for critical neuromuscular research

Forty-two medical researchers and their labs have been awarded over $21 million by the Muscular Dystrophy Association to continue critical neuromuscular research. Many of the grants are multi-year awards to be dispersed over the next three years.

1 Apr 2010

Scientists link gene expression profiling effort to identify potential novel therapeutic for ALS

In an article published in Nature Genetics today, scientists at the ALS Therapy Development Institute describe how they have linked an unbiased gene expression profiling effort to the identification and successful preclinical testing of a potential novel therapeutic for amyotrophic lateral sclerosis.

29 Mar 2010