Myopathy News and Research RSS Feed - Myopathy News and Research

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).

Researchers show that vitamin B3 can slow down progression of mitochondrial disease

The researchers of the University of Helsinki, Finland, and école Polytechnique Fédérale de Lausanne, Switzerland, have shown that vitamin B3 form nicotinamide riboside can slow down the progression of mitochondrial disease, suggesting its potential as a novel therapy approach to adult-onset mitochondrial muscle diseases. [More]

Jain Foundation's dysferlinopathy clinical study exceeds original goal of 150 patients

The Jain Foundation is delighted to announce that 193 patients have been recruited into the Clinical Outcome Study for Dysferlinopathy (COS), exceeding the original goal of 150 patients. [More]
Investigators urge drug regulators to provide clear evidence on statins’ side effects

Investigators urge drug regulators to provide clear evidence on statins’ side effects

At a time when the wider prescription of statins is under renewed public scrutiny, a substantial analysis of placebo-controlled randomised trials of statins has found that only a small minority of side effects reported by those taking the cholesterol-lowering drugs are actually attributable to them. [More]
Patients with congenital disorders are urged to register with CMDIR to advance clinical trials

Patients with congenital disorders are urged to register with CMDIR to advance clinical trials

If you are affected by Malignant Hyperthermia, Congenital Muscular Dystrophies or Congenital Myopathy Subtypes, the Malignant Hyperthermia Association of the United States and the Cure Congenital Muscle Disease organization are asking for your help to enable research and clinical trials by registering with the Congenital Muscle Disease International Registry (CMDIR). [More]
Study: Genetic mutations in titin gene can cause skeletal muscle disease

Study: Genetic mutations in titin gene can cause skeletal muscle disease

A University of Arizona doctoral candidate has shown for the first time that genetic mutations in the titin gene can cause skeletal muscle myopathy, a disease in which muscle fibers do not function properly, resulting in muscle weakness. [More]
Merck announces FDA approval of ISENTRESS for oral suspension

Merck announces FDA approval of ISENTRESS for oral suspension

Merck, known as MSD outside the United States and Canada, announced today that the U.S. Food and Drug Administration recently approved ISENTRESS for oral suspension, a new pediatric formulation of Merck's integrase inhibitor. With this approval, ISENTRESS is now indicated in combination with other antiretroviral agents for the treatment of HIV-1 infection in patients four weeks of age and older. [More]
New gene mutation helps doctors give more accurate diagnosis of mitochondrial myopathy

New gene mutation helps doctors give more accurate diagnosis of mitochondrial myopathy

A new gene mutation which will help doctors give a more accurate diagnosis of a particular type of brain and muscle disease in children has been discovered for the first time by University of Leeds experts. [More]
Soligenix initiates orBec Phase 2 study for GI manifestations of chronic GVHD

Soligenix initiates orBec Phase 2 study for GI manifestations of chronic GVHD

Soligenix, Inc., a clinical stage biopharmaceutical company focused on developing products to treat serious inflammatory diseases where there remains an unmet medical need, as well as developing several biodefense vaccines and therapeutics, announced today that it has initiated a Phase 2, randomized, double-blind, placebo-controlled study evaluating orBec (oral beclomethasone 17,21-dipropionate or BDP) as a treatment for the gastrointestinal manifestations of chronic Graft-versus-Host disease. [More]
Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

... [More]

Merck to present pharmacokinetic data on once-daily ISENTRESS at EACS

Merck, known as MSD outside the United States and Canada, is presenting pharmacokinetic data this week on investigational formulations of a once daily dose of ISENTRESS at the 14th European AIDS Conference, sponsored by the European AIDS Clinical Society. [More]

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

... [More]
Four new pre-clinical drug development projects at NIH develop treatment for rare disease

Four new pre-clinical drug development projects at NIH develop treatment for rare disease

Four new pre-clinical drug development projects at the National Institutes of Health will target a form of blindness and diseases characterized by cardiac problems. The projects were selected for their potential to treat specific rare diseases and to help scientists uncover new information that can be shared with other researchers. [More]

Evotec And Jain Extend Research Collab In Skeletal Muscular Dystrophy

... [More]
Genetic variations in genes could put patients at risk for statin-associated muscle injury: Study

Genetic variations in genes could put patients at risk for statin-associated muscle injury: Study

Statins, a class of drugs used to lower cholesterol, are among the best selling drugs in North America and around the world. However, statin myopathy, which results in muscle pain and weakness, is a common side effect affecting up to 10 percent of statin users. A recent study led by Dr. Richard Kim of the Lawson Health Research Institute, in collaboration with Dr. Robert Hegele of Robarts Research Institute, and researchers from Vanderbilt University, found that commonly occurring genetic variations in a person's genes could put them at risk for statin-associated muscle injury. [More]

Audentes Therapeutics closes $30 million Series A financing

Audentes Therapeutics, Inc., a recently founded biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, today announced the closing of a $30 million Series A financing. [More]
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

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]