Dystrophin News and Research

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Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.
New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy

New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy

Genethon announces dosing of first patient at pediatric clinical trial platform for neuromuscular diseases

Genethon announces dosing of first patient at pediatric clinical trial platform for neuromuscular diseases

Stem cell therapy could help restore debilitating age-related muscle loss

Stem cell therapy could help restore debilitating age-related muscle loss

FDA approval provides targeted treatment option for patients with rare DMD mutation

FDA approval provides targeted treatment option for patients with rare DMD mutation

Study unlocks some muscle cell secrets

Study unlocks some muscle cell secrets

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Genetic mutation may accelerate heart function decline in DMD patients

Genetic mutation may accelerate heart function decline in DMD patients

NIH awards grants for using bioengineered models to improve clinical trial design

NIH awards grants for using bioengineered models to improve clinical trial design

New drug offers hope for young boys with Duchenne muscular dystrophy

New drug offers hope for young boys with Duchenne muscular dystrophy

Study uncovers a promising new therapeutic approach for Duchenne muscular dystrophy

Study uncovers a promising new therapeutic approach for Duchenne muscular dystrophy

Researchers find link between dystrophic muscles and lymphatic system in mice with Duchenne disease

Researchers find link between dystrophic muscles and lymphatic system in mice with Duchenne disease

FDA approves new treatment option for patients with rare Duchenne muscular dystrophy mutation

FDA approves new treatment option for patients with rare Duchenne muscular dystrophy mutation

Micro-dystrophin gene therapy can improve outcomes in children with Duchenne muscular dystrophy

Micro-dystrophin gene therapy can improve outcomes in children with Duchenne muscular dystrophy

UTSW/Children's Health joint program approved as Certified Duchenne Care Center

UTSW/Children's Health joint program approved as Certified Duchenne Care Center

New study indicates novel therapies for Duchenne muscular dystrophy

New study indicates novel therapies for Duchenne muscular dystrophy

Model that sheds light on severe Duchenne muscular dystrophy could pave the way for new therapies

Model that sheds light on severe Duchenne muscular dystrophy could pave the way for new therapies

Small molecules may open the door to new therapies for Duchenne muscular dystrophy

Small molecules may open the door to new therapies for Duchenne muscular dystrophy

Study reveals molecular pathogenesis of muscular dystrophy-associated cardiomyopathy

Study reveals molecular pathogenesis of muscular dystrophy-associated cardiomyopathy

New gene correction therapy for hereditary muscular disease among children

New gene correction therapy for hereditary muscular disease among children

PerkinElmer provides newborn screening assay for PPMD’s New York State pilot program

PerkinElmer provides newborn screening assay for PPMD’s New York State pilot program