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.
AMT receives Innovation Credit to develop gene therapy treatment for DMD

AMT receives Innovation Credit to develop gene therapy treatment for DMD

Humans and mice have critical differences in gene responsible for DMD

Humans and mice have critical differences in gene responsible for DMD

AMT's gene therapy successful in treating Duchenne muscular dystrophy

AMT's gene therapy successful in treating Duchenne muscular dystrophy

Research shows exon-skipping drug may prove effective for treating Duchenne muscular dystrophy

Research shows exon-skipping drug may prove effective for treating Duchenne muscular dystrophy

Amsterdam Molecular Therapeutics' AMT-080 granted EMEA Orphan Drug Designation

Amsterdam Molecular Therapeutics' AMT-080 granted EMEA Orphan Drug Designation

UCLA discovery raises hope for curing genetic disorders caused by nonsense mutations

UCLA discovery raises hope for curing genetic disorders caused by nonsense mutations

Charley's Fund and the Nash Avery Foundation to support investigation of Galapagos' SARM candidate drug

Charley's Fund and the Nash Avery Foundation to support investigation of Galapagos' SARM candidate drug

AVI BioPharma's peptide-conjugated PMO chemistry to be discussed at two upcoming scientific meetings

AVI BioPharma's peptide-conjugated PMO chemistry to be discussed at two upcoming scientific meetings

New function for missing  protein in Duchenne muscular dystrophy found

New function for missing protein in Duchenne muscular dystrophy found

New therapy shows potential for treating Duchenne muscular dystrophy

New therapy shows potential for treating Duchenne muscular dystrophy

Inflammation clue to fragile bones in Duchenne's muscular dystrophy

Inflammation clue to fragile bones in Duchenne's muscular dystrophy

First treatment for muscular dystrophy a step closer

First treatment for muscular dystrophy a step closer

Novel genetic technology for canine form of muscular dystrophy

Novel genetic technology for canine form of muscular dystrophy

Discovery of stem cells that repair injured muscles

Discovery of stem cells that repair injured muscles

New findings raise questions about process used to identify experimental drug

New findings raise questions about process used to identify experimental drug

Proteins called ankyrins required to prevent muscular dystrophy

Proteins called ankyrins required to prevent muscular dystrophy

Adding protein sarcospan to muscle cells might help Duchenne muscular dystrophy

Adding protein sarcospan to muscle cells might help Duchenne muscular dystrophy

New type of gene manipulation therapy offers hope for Duchenne muscular dystrophy sufferers

New type of gene manipulation therapy offers hope for Duchenne muscular dystrophy sufferers

Proof-of-concept that purified muscle stem cells can be used in therapy

Proof-of-concept that purified muscle stem cells can be used in therapy

Investigational drug Debio-025 tested for preventing muscle fiber death in muscular dystrophy

Investigational drug Debio-025 tested for preventing muscle fiber death in muscular dystrophy