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 breakthrough may provide key to understanding how to treat Muscular Dystrophy

New breakthrough may provide key to understanding how to treat Muscular Dystrophy

Tarix Orphan's TXA127 granted FDA Fast Track Designation for treatment of DMD patients

Tarix Orphan's TXA127 granted FDA Fast Track Designation for treatment of DMD patients

New gene therapy approach to treating DMD reduces symptoms, extends life span in mouse model

New gene therapy approach to treating DMD reduces symptoms, extends life span in mouse model

PTC Therapeutics announces recipients of STRIVE Awards

PTC Therapeutics announces recipients of STRIVE Awards

New iPS cell model provides clear drug target for treatment of DMD

New iPS cell model provides clear drug target for treatment of DMD

Kay E. Davies named recipient of ASHG's 2015 William Allan Award

Kay E. Davies named recipient of ASHG's 2015 William Allan Award

University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

Duke researchers demonstrate genetic therapeutic technique to treat DMD patients

Duke researchers demonstrate genetic therapeutic technique to treat DMD patients

UI Children's Hospital named Certified Duchenne Care Center by PPMD

UI Children's Hospital named Certified Duchenne Care Center by PPMD

Study demonstrates therapeutic potential of new class of synthetic oligonucleotides for DMD treatment

Study demonstrates therapeutic potential of new class of synthetic oligonucleotides for DMD treatment

Bern researchers develop active substance for treating Duchenne muscular dystrophy

Bern researchers develop active substance for treating Duchenne muscular dystrophy

Early use of heart failure drugs slows cardiac damage in boys, young men with DMD

Early use of heart failure drugs slows cardiac damage in boys, young men with DMD

PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC begins rolling NDA submission for Translarna to treat nmDMD

Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

PPMD, Santhera Pharmaceuticals collaborate to study Duchenne muscular dystrophy

PPMD, Santhera Pharmaceuticals collaborate to study Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Research offers hope to patient suffering from Duchenne muscular dystrophy

Research offers hope to patient suffering from Duchenne muscular dystrophy

Isoform: New protein offers novel therapeutic approach for patients with DMD

Isoform: New protein offers novel therapeutic approach for patients with DMD

PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics initiates reimbursed expanded access program for Translarna