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.

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

Dementia diagnostic panel for treatable forms of cognitive impairment launched by Quest Diagnostics

Quest Diagnostics (NYSE: DGX), the world’s leading provider of diagnostic information services, announced the availability of the first clinical test panel for aiding the diagnosis of suspected dementia due to treatable forms of cognitive impairment. The test panel is believed to be the first commercial service from a clinical laboratory to combine several guideline-recommended tests for identifying secondary, treatable causes of dementia as a single blood test and report.

From Quest Diagnostics 19 Mar 2013

Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

The following research from Columbia University Medical Center and NewYork-Presbyterian Hospital is being presented at the 65th annual meeting of the American Academy of Neurology, March 16-23, 2013, in San Diego.

18 Mar 2013

Researchers identify new mechanism in onset of motor neuron diseases

The British astrophysicist Stephen Hawking is likely to be the world's most famous person living with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. ALS is a progressive disease affecting motor neurons, nerve cells that control muscle function, and nearly always leads to death.

11 Mar 2013

Genetic mutation that alters PIEZO1 ion channel in RBCs contributes to familial xerocytosis

A genetic mutation that alters the kinetics of an ion channel in red blood cells has been identified as the cause behind a hereditary anemia, according to a paper published this month in the Proceedings of the National Academy of Sciences by University at Buffalo scientists and colleagues.

11 Mar 2013

Cell fusion studies at Johns Hopkins could lead to improved treatments for muscular dystrophy

Researchers at Johns Hopkins have established a high-efficiency cell-cell fusion system, providing a new model to study how fusion works. The scientists showed that fusion between two cells is not equal and mutual as some assumed, but, rather, is initiated and driven by one of the fusion partners. The discovery, they say, could lead to improved treatments for muscular dystrophy, since muscle regeneration relies on cell fusion to make muscle fibers that contain hundreds or even thousands of nuclei.

8 Mar 2013

Study combines induced pluripotent stem cell technology and genetic correction to treat DMD

Researchers at the University of Minnesota's Lillehei Heart Institute have combined genetic repair with cellular reprogramming to generate stem cells capable of muscle regeneration in a mouse model for Duchenne Muscular Dystrophy (DMD).

7 Mar 2013

Pivotal study of olesoxime in Spinal Muscular Atrophy receives positive interim review

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for under-served medical needs in neurology and cardiology, announced the completion of the interim analysis of the pivotal efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA).

1 Mar 2013

New findings could help develop treatments for inherited disorders

Fresh insights into the protective seal that surrounds the DNA of our cells could help develop treatments for inherited muscle, brain, bone and skin disorders.

27 Feb 2013

New approach could make gene therapy dramatically more effective for HIV patients

A research team from Case Western Reserve University School of Medicine has discovered an approach that could make gene therapy dramatically more effective for patients.

16 Feb 2013

Steroid therapy impedes Duchenne’s cardiomyopathy

Steroid therapy is associated with a considerable reduction in all-cause mortality and new-onset and progressive cardiomyopathy in patients with the debilitating X-linked disease Duchenne muscular dystrophy.

11 Feb 2013

Left ventricular assist device improves quality of life in two brothers with Becker's Muscular Dystrophy

Tyler Wertz, 18, and his 16-year old brother Austin Tucker, fight, tease, love and support each other like most brothers do. Unlike most brothers, however, they share the same uncertain future—both have heart assist devices keeping them alive while they struggle with a debilitating disease. As young boys, Tyler and Austin were diagnosed with Becker's Muscular Dystrophy, a disease that slowly weakens the arms and legs and damages the heart.

31 Jan 2013

CUMC receives $7M to help establish Weinberg Family Cerebral Palsy Center

Seeking to bridge the transition from pediatric to adult care for people living with cerebral palsy, Debby and Peter A. Weinberg, with several of their family members and friends, have given more than $7 million to help establish the Weinberg Family Cerebral Palsy Center at Columbia University Medical Center (CUMC).