Myopathies News and Research

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Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

Researchers discover two proteins essential for development of skeletal muscle

Researchers discover two proteins essential for development of skeletal muscle

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

Study provides vital information to develop successful PI5P4K inhibitors for p53-mutant cancers

Study provides vital information to develop successful PI5P4K inhibitors for p53-mutant cancers

Removal of single mitochondrial protein leads to severe inflammation

Removal of single mitochondrial protein leads to severe inflammation

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

New research offers crucial step towards understanding cause of myopathy

New research offers crucial step towards understanding cause of myopathy

Researchers discover small protein essential for formation of skeletal muscle

Researchers discover small protein essential for formation of skeletal muscle

Some WADA-banned substances may have potential medical applications

Some WADA-banned substances may have potential medical applications

Key gene controls ability of adult stem cells to regenerate muscle after injury, study finds

Key gene controls ability of adult stem cells to regenerate muscle after injury, study finds

Researchers investigate biochemical, physiological characteristics of facial and extraocular muscles

Researchers investigate biochemical, physiological characteristics of facial and extraocular muscles

Berlin researchers identify defects in myotubular myopathy

Berlin researchers identify defects in myotubular myopathy

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

PTC Therapeutics announces recipients of STRIVE Awards

PTC Therapeutics announces recipients of STRIVE Awards

Necrotising autoimmune myopathy requires early, aggressive treatment

Necrotising autoimmune myopathy requires early, aggressive treatment

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

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

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

Researchers review ten years of scientific studies on mitochondrial toxicity in pregnant women

Researchers review ten years of scientific studies on mitochondrial toxicity in pregnant women

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

Bern researchers develop active substance for treating Duchenne muscular dystrophy

Bern researchers develop active substance for treating Duchenne muscular dystrophy