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The myopathies are neuromuscular disorders in which the primary symptom is muscle weakness due to dysfunction of muscle fiber. Other symptoms of myopathy can include include muscle cramps, stiffness, and spasm. Myopathies can be inherited (such as the muscular dystrophies) or acquired (such as common muscle cramps).
Merck's LIPTRUZET tablets get FDA approval for treatment of elevated LDL cholesterol

Merck's LIPTRUZET tablets get FDA approval for treatment of elevated LDL cholesterol

Merck, known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration has approved LIPTRUZET (ezetimibe and atorvastatin) tablets for the treatment of elevated low-density lipoprotein cholesterol in patients with primary or mixed hyperlipidemia as adjunctive therapy to diet when diet alone is not enough. [More]
HUGO recognizes Dr. Patrick Tan for research on genomic profiles of Asian cancers

HUGO recognizes Dr. Patrick Tan for research on genomic profiles of Asian cancers

Dr Patrick Tan from A*STAR's Genome Institute of Singapore has received the 2013 Chen New Investigator Award from the international Human Genome Organisation. [More]
Genetic testing services for rare neurological disorders launched by Athena Diagnostics

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. [More]
Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

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. [More]
VCP gene mutations cause neurological conditions including IBMPFD and ALS

VCP gene mutations cause neurological conditions including IBMPFD and ALS

New research, published in Neuron, gives insight into how single mutations in the VCP gene cause a range of neurological conditions including a form of dementia called Inclusion Body Myopathy, Paget's Disease of the Bone and Frontotemporal Dementia (IBMPFD), and the motor neuron disease Amyotrophic Lateral Sclerosis (ALS). [More]
Study identifies how new ALS gene mutations cause other diseases of the brain, muscle and bone

Study identifies how new ALS gene mutations cause other diseases of the brain, muscle and bone

Researchers supported by The ALS Association have discovered how mutations in new amyotrophic lateral sclerosis (ALS) genes cause not only ALS but also other diseases of the brain, muscle and bone. [More]
Selenium supplements and heart disease risk: an interview with Dr Saverio Stranges, University of Warwick

Selenium supplements and heart disease risk: an interview with Dr Saverio Stranges, University of Warwick

Selenium is a micronutrient which has antioxidant properties by means of selenoproteins which play many important functions in the body. They actually are contained in many different foods. The amount of selenium we get from diet is closely linked to the amount of selenium in the soil. [More]
Clinical trial to evaluate cyclodextrin against Niemann-Pick disease type C1

Clinical trial to evaluate cyclodextrin against Niemann-Pick disease type C1

A clinical trial to evaluate a drug candidate called cyclodextrin as a possible treatment for Niemann-Pick disease type C1 (NPC), a rare and fatal genetic disease, will start today, researchers announced. [More]

New targeted enzyme replacement therapy can treat X-linked myotubular myopathy

A collaborative research team including a Medical College of Wisconsin (MCW) pediatric neuropathologist successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy from 4s3 Bioscience. [More]
Merck’s resubmission of the NDA for suggammadex sodium injection accepted for review by the FDA

Merck’s resubmission of the NDA for suggammadex sodium injection accepted for review by the FDA

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the resubmission of the New Drug Application (NDA) for sugammadex sodium injection has been accepted for review by the U.S. Food and Drug Administration (FDA). Merck expects the FDA’s review to be completed in the first half of 2013. [More]

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength. [More]
Longer looks: People with mental disabilities have a vote; Genome sequencing raises hope - also anxiety

Longer looks: People with mental disabilities have a vote; Genome sequencing raises hope - also anxiety

I may get Alzheimer's disease, I was told after getting my DNA analyzed. I could suffer the consequences of an abnormal blood clot in my heart or my brain. Or perhaps I'll suffer from a disease called primary myelofibrosis that's caused by an overactive bone marrow. All of these were possible outcomes, based on my genetic profile. In each case, the test only gives an increased risk for disease, not a definitive diagnosis. [More]
‘Gene for speed’ researcher awarded Ramaciotti Medal

‘Gene for speed’ researcher awarded Ramaciotti Medal

Professor Kathryn North AM, the researcher responsible for discovering the ‘gene for speed’, has been awarded the prestigious Ramaciotti Medal for Excellence in Biomedical Research and a $50,000 grant. [More]
Intensive treatment with high doses of statin drugs shows promise for dyslipidemia

Intensive treatment with high doses of statin drugs shows promise for dyslipidemia

A comprehensive new review on how to treat high cholesterol and other blood lipid problems suggests that intensive treatment with high doses of statin drugs is usually the best approach. [More]
New mouse model could foster ALS, Paget's and dementia breakthroughs

New mouse model could foster ALS, Paget's and dementia breakthroughs

Efforts to treat disorders like Lou Gehrig's disease, Paget's disease, inclusion body myopathy and dementia will receive a considerable boost from a new research model created by UC Irvine scientists. [More]

New insights into filaminopathies

New insights into certain muscle diseases, the filaminopathies, are reported by an international research team led by Dr. Rudolf Andre Kley of the RUB's University Hospital Bergmannsheil in the journal Brain. [More]

New clinical trial to evaluate DEX-M74 against hereditary inclusion body myopathy

Researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM). National Institutes of Health scientists from the National Center for Advancing Translational Sciences (NCATS) and the National Human Genome Research Institute (NHGRI) will conduct the clinical trial at the NIH Clinical Center. [More]
NCI awards Soligenix $300,000 SBIR grant for orBec Phase 2 chronic GI GVHD trial

NCI awards Soligenix $300,000 SBIR grant for orBec Phase 2 chronic GI GVHD trial

Soligenix, Inc., a development stage biopharmaceutical company, announced today that the National Cancer Institute has awarded Soligenix a Small Business Innovation Research grant to support the conduct of a Phase 2 clinical trial designed to evaluate orBec (oral beclomethasone 17,21-dipropionate or BDP) as a treatment for the gastrointestinal manifestation of chronic Graft-versus-Host disease. [More]
Statin's potency may cause muscle problems in patients with cardiovascular disease

Statin's potency may cause muscle problems in patients with cardiovascular disease

A study from the University of California, San Diego School of Medicine, published August 22 online by PLoS ONE, reports that muscle problems reported by patients taking statins were related to the strength or potency of the given cholesterol-lowering drugs. [More]
Researchers discover new cause of congenital myopathy

Researchers discover new cause of congenital myopathy

University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics. [More]