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.

Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

A research team at Cold Spring Harbor Laboratory has used a recently developed technology they call TSUNAMI to create the first animal model of the adult-onset version of spinal muscular atrophy, a devastating motor-neuron illness.

10 Sep 2013

Case Western researchers establish Kruppel-like factors as master regulators of vascular health

Case Western Reserve researchers have identified a genetic factor that blocks the blood vessel inflammation that can lead to heart attacks, strokes and other potentially life-threatening events.

5 Sep 2013

Repligen Earns $1M Pfizer Milestone Payment

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4 Sep 2013

Scientists identify specific type of receptor that may aid in treatment of AD, Parkinson’s disease

Scientists at Washington University School of Medicine in St. Louis have found a way that corrupted, disease-causing proteins spread in the brain, potentially contributing to Alzheimer's disease, Parkinson's disease and other brain-damaging disorders.

26 Aug 2013

Evotec And Jain Extend Research Collab In Skeletal Muscular Dystrophy

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22 Aug 2013

Gene combination therapy repairs and prevents future damage of muscles caused by DMD

New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease.

12 Aug 2013

Study: 3 enzymes required for building sugar superstructure involved in muscular dystrophies

For many inherited diseases, such as cystic fibrosis or Huntington disease, the disease-causing genetic mutation damages or removes a protein that has an essential role in the body. This protein defect is the root cause of the disease symptoms.

9 Aug 2013

Eteplirsen offers great hope to patients with Duchenne muscular dystrophy

Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health.

8 Aug 2013

Trace substance in sugar improves muscle regeneration in mouse model with muscular dystrophy

A trace substance in caramelized sugar, when purified and given in appropriate doses, improves muscle regeneration in a mouse model of Duchenne muscular dystrophy. The findings are published today, Aug. 1, in the journal Skeletal Muscle.

2 Aug 2013

Study results suggest reexamination of biological, medical importance of X chromosome

Painstaking new analysis of the genetic sequence of the X chromosome—long perceived as the "female" counterpart to the male-associated Y chromosome—reveals that large portions of the X have evolved to play a specialized role in sperm production.

24 Jul 2013

3D-printed airway splint saves life of infant born with severe tracheobronchomalacia

Half a millennium after Johannes Gutenberg printed the bible, researchers printed a 3D splint that saved the life of an infant born with severe tracheobronchomalacia, a birth defect that causes the airway to collapse.

12 Jul 2013

TSRI scientists identify small molecules to control genetic defect responsible for muscular dystrophy

For the first time, scientists from the Florida campus of The Scripps Research Institute have identified small molecules that allow for complete control over a genetic defect responsible for the most common adult onset form of muscular dystrophy.

28 Jun 2013

A paradigm shift for treatment of muscular dystrophy

Researchers at Boston University College of Health & Rehabilitation Sciences: Sargent College have identified a combinatorial therapeutic approach that has proven effective in treating muscular dystrophy in a mouse model.

26 Jun 2013

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