Myopathy News and Research

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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 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.

9 Jan 2014

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.

17 Dec 2013

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.

2 Dec 2013

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

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17 Oct 2013

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.

17 Oct 2013

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

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16 Oct 2013

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.

13 Sep 2013

Evotec And Jain Extend Research Collab In Skeletal Muscular Dystrophy

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22 Aug 2013

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.

7 Aug 2013

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.

18 Jul 2013

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.

4 May 2013

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.

19 Apr 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

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

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).

14 Mar 2013

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.

4 Mar 2013

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.

21 Feb 2013

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.

24 Jan 2013

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.

23 Jan 2013

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.

7 Jan 2013

Myopathy News and Research

Further Reading

Merck announces FDA approval of ISENTRESS for oral suspension

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

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

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

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

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

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

Evotec And Jain Extend Research Collab In Skeletal Muscular Dystrophy

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

Audentes Therapeutics closes $30 million Series A financing

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

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

Genetic testing services for rare neurological disorders launched by Athena Diagnostics

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

VCP gene mutations cause neurological conditions including IBMPFD and ALS

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

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

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

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

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

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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