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 $12.7M to research groups to explore new treatments for patients in eight disease areas

The National Institutes of Health has awarded $12.7 million to match nine academic research groups with a selection of pharmaceutical industry compounds to explore new treatments for patients in eight disease areas, including Alzheimer's disease, Duchenne muscular dystrophy and schizophrenia.

19 Jun 2013

New drug target may prevent consequences of ACL tear in athletes

Striking the likes of Chicago Bulls' Derrick Rose, L.A. Lakers' Kobe Bryant and Detroit Tigers' Victor Martinez, tears in the anterior cruciate ligament (ACL) are one of the most rampant and serious knee injuries among athletes.

14 Jun 2013

Slowdowns in transport of important molecules may contribute to development of ALS

Slowdowns in the transport and delivery of nutrients, proteins and signaling molecules within nerve cells may contribute to the development of the neurodegenerative disorder ALS, according to researchers at the University of Illinois at Chicago College of Medicine.

13 Jun 2013

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients.

5 Jun 2013

Cedars-Sinai team awarded grant to study cardiac stem cell treatment for Duchenne muscular dystrophy

Newport Beach-based nonprofit Coalition Duchenne has awarded a $150,000 grant to a Cedars-Sinai Heart Institute team investigating whether an experimental cardiac stem cell treatment could be used to treat Duchenne muscular dystrophy patients who have developed heart disease.

30 May 2013

Researchers find a way the body can remove injured axons

Many medical issues affect nerves, from injuries in car accidents and side effects of chemotherapy to glaucoma and multiple sclerosis. The common theme in these scenarios is destruction of nerve axons, the long wires that transmit signals to other parts of the body, allowing movement, sight and sense of touch, among other vital functions.

10 May 2013

Study offers new hope to patients suffering from laminopathies

Laminopathies are hereditary diseases that affect mainly the muscle tissue. These diseases include for example Emery-Dreifuss Muscular dystrophy, dilated cardiomyopathy, limb-girdle muscular dystrophy and Hutchison-Gilford progeria syndrome.

7 May 2013

New hope for patients with myotonic dystrophy

There's hope for patients with myotonic dystrophy. A new small molecule developed by researchers at the University of Illinois has been shown to break up the protein-RNA clusters that cause the disease in living human cells, an important first step toward developing a pharmaceutical treatment for the as-yet untreatable disease.

2 May 2013

Study: Melatonin delays symptom onset, reduces mortality in mouse model of ALS

Melatonin injections delayed symptom onset and reduced mortality in a mouse model of the neurodegenerative condition amyotrophic lateral sclerosis, or Lou Gehrig's disease, according to a new study by researchers at the University of Pittsburgh School of Medicine.

25 Apr 2013

Investigational treatment for ALS passes early phase clinical trial for safety

An investigational treatment for an inherited form of Lou Gehrig's disease has passed an early phase clinical trial for safety, researchers at Washington University School of Medicine in St. Louis and Massachusetts General Hospital report.

24 Apr 2013

Isis Pharmaceuticals starts ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.

24 Apr 2013

Study highlights importance of zebrafish as a model organism for human disease research

Researchers demonstrate today that 70 per cent of protein-coding human genes are related to genes found in the zebrafish and that 84 per cent of genes known to be associated with human disease have a zebrafish counterpart.

18 Apr 2013

ARMGO Pharma gets $1 million to support ARM210 preclinical work for DMD treatment

ARMGO Pharma and the Muscular Dystrophy Association today announced that $1 Million has been awarded for preclinical work in support of an Investigational New Drug application with the U.S. Food and Drug Administration for ARM210, a novel, orally available, small-molecule Rycal drug that has potential as a treatment for Duchenne muscular dystrophy.

18 Apr 2013

Johns Hopkins scientist awarded Ho-Am prize for discovery of myostatin

South Korea's Ho-Am Foundation announced that Johns Hopkins researcher Se-Jin Lee, M.D., Ph.D., has won this year's Ho-Am Prize in Medicine.

5 Apr 2013

Clinical trial signals new era in treatment of neurodegererative disorders

The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis - blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease - may be a first step towards a new era in the treatment of such disorders.

4 Apr 2013

Exosomes can transfer membrane proteins, study reveals

Important new research from UMass Medical School demonstrates how exosomes shuttle proteins from neurons to muscle cells where they take part in critical signaling mechanisms, an exciting discovery that means these tiny vehicles could one day be loaded with therapeutic agents, such as RNA interference, and directly target disease-carrying cells.

28 Mar 2013

Royal Holloway University supports Muscular Dystrophy Campaign

Royal Holloway University, is today giving money generated by its research into Duchenne muscular dystrophy to the Muscular Dystrophy Campaign.

27 Mar 2013

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy, announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV.

21 Mar 2013

Research findings could lead to development of new drugs for gout treatment

Findings from a Loyola University Chicago Stritch School of Medicine study could lead to the development of new drugs to treat gout.

20 Mar 2013

Genetic testing services for rare neurological disorders launched by Athena Diagnostics

Athena Diagnostics, a leader in neurological diagnostics, today announced the clinical availability of new genetic tests to aid the detection of several rare neurological disorders, including hereditary neuropathy, neuromuscular disease, epilepsy and certain movement disorders. The lab-developed tests are available through Athena Diagnostics, a business of Quest Diagnostics (NYSE:DGX), the world’s leading provider of diagnostic information services.

From Quest Diagnostics 19 Mar 2013