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.
Experimental treatment shows early promise to help nearly half of DMD patients

Experimental treatment shows early promise to help nearly half of DMD patients

Cheaper drug equally effective in preserving cardiovascular function in boys with DMD

Cheaper drug equally effective in preserving cardiovascular function in boys with DMD

Study: Gene editing may correct genetic mutation responsible for Duchenne muscular dystrophy

Study: Gene editing may correct genetic mutation responsible for Duchenne muscular dystrophy

New drug provides hope for patients with Duchenne muscular dystrophy

New drug provides hope for patients with Duchenne muscular dystrophy

New discovery could improve therapies for Duchenne muscular dystrophy

New discovery could improve therapies for Duchenne muscular dystrophy

Novo Biosciences achieves major milestones in its new drug to treat heart disease and DMD

Novo Biosciences achieves major milestones in its new drug to treat heart disease and DMD

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Johns Hopkins researchers find genetic cause of olfactory neuroblastoma

Johns Hopkins researchers find genetic cause of olfactory neuroblastoma

Clinical study shows tolerability and safety of investigational drug in boys with DMD

Clinical study shows tolerability and safety of investigational drug in boys with DMD

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Single CRISPR treatment can safely and stably correct genetic disease

Single CRISPR treatment can safely and stably correct genetic disease

Study findings hold promise for children with DMD

Study findings hold promise for children with DMD

Researchers identify and overcome barrier in CRISPR gene editing to treat muscular dystrophy

Researchers identify and overcome barrier in CRISPR gene editing to treat muscular dystrophy

Urine exRNA may be source of biomarkers for muscular dystrophy

Urine exRNA may be source of biomarkers for muscular dystrophy

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Muscle dystrophy treatment in dogs with Crispr gene editing

Muscle dystrophy treatment in dogs with Crispr gene editing

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

New project to combat DMD-related fibrosis receives major funding boost

New project to combat DMD-related fibrosis receives major funding boost

New cell-based technologies could help improve understanding of muscle-wasting disease

New cell-based technologies could help improve understanding of muscle-wasting disease

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy