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

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

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

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

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

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

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

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

Audentes Therapeutics closes $30 million Series A financing

Audentes Therapeutics closes $30 million Series A financing

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

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

Genetic testing services for rare neurological disorders launched by Athena Diagnostics

Genetic testing services for rare neurological disorders launched by Athena Diagnostics

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

VCP gene mutations cause neurological conditions including IBMPFD and ALS

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

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

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

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

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

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

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

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

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