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).
Researchers unlock structural details of key protein to treat neuromuscular disease

Researchers unlock structural details of key protein to treat neuromuscular disease

Research highlights potential new treatment strategy for degenerative diseases

Research highlights potential new treatment strategy for degenerative diseases

First patient dosed in long term-safety extension study of Resolaris for adult patients with FSHD

First patient dosed in long term-safety extension study of Resolaris for adult patients with FSHD

Scientists reveal role of dynamin and show how specific mutations impair function of dynamin

Scientists reveal role of dynamin and show how specific mutations impair function of dynamin

Clementia expands enrollment to include children with FOP in ongoing Phase 2 clinical trial

Clementia expands enrollment to include children with FOP in ongoing Phase 2 clinical trial

Necrotising autoimmune myopathy requires early, aggressive treatment

Necrotising autoimmune myopathy requires early, aggressive treatment

Substantial pharmacogenetic information available on cardiovascular drugs

Substantial pharmacogenetic information available on cardiovascular drugs

Clementia Pharmaceuticals commences multi-center study of patients with fibrodysplasia ossificans progressiva

Clementia Pharmaceuticals commences multi-center study of patients with fibrodysplasia ossificans progressiva

aTyr's Resolaris granted FDA Orphan Drug Designation for treatment of FSHD

aTyr's Resolaris granted FDA Orphan Drug Designation for treatment of FSHD

Joseph Roche receives grant to develop improved exercise programs for people with dysferlinopathies

Joseph Roche receives grant to develop improved exercise programs for people with dysferlinopathies

TGen scientists discover the likely cause of rare type of muscle weakness in six children

TGen scientists discover the likely cause of rare type of muscle weakness in six children

EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

Clementia secures additional $10 million to support development of palovarotene for treatment of FOP

Clementia secures additional $10 million to support development of palovarotene for treatment of FOP

AutoGenomics, Genomas sign agreement to develop DNA-guided diagnostic systems

AutoGenomics, Genomas sign agreement to develop DNA-guided diagnostic systems

Merck enrolls first patient in reformulated raltegravir Phase 3 trial for treatment of HIV-1 infection

Merck enrolls first patient in reformulated raltegravir Phase 3 trial for treatment of HIV-1 infection

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

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

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

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

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

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

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

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

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