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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
Royal Holloway University, is today giving money generated by its research into Duchenne muscular dystrophy to the Muscular Dystrophy Campaign.
[More]
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.
[More]
Findings from a Loyola University Chicago Stritch School of Medicine study could lead to the development of new drugs to treat gout.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]
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.
[More]