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.
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

FDA grants Fast Track designation to Akashi Therapeutics' HT-100 for treatment of DMD

FDA grants Fast Track designation to Akashi Therapeutics' HT-100 for treatment of DMD

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

Commonly used drug restores blood flow to oxygen-starved muscles of boys with muscular dystrophy

Commonly used drug restores blood flow to oxygen-starved muscles of boys with muscular dystrophy

Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

Sarepta Therapeutics plans to submit eteplirsen NDA for treatment of Duchenne muscular dystrophy

Sarepta Therapeutics plans to submit eteplirsen NDA for treatment of Duchenne muscular dystrophy

Sarepta Therapeutics reports non-GAAP net loss of $29.1 million for Q4 2013

Sarepta Therapeutics reports non-GAAP net loss of $29.1 million for Q4 2013

Researchers demonstrate new approach to treating muscular dystrophy

Researchers demonstrate new approach to treating muscular dystrophy

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Three Penn Medicine researchers awarded grants for neuromuscular disease research

Three Penn Medicine researchers awarded grants for neuromuscular disease research

VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy

VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy

Gene combination therapy repairs and prevents future damage of muscles caused by DMD

Gene combination therapy repairs and prevents future damage of muscles caused by DMD

Eteplirsen offers great hope to patients with Duchenne muscular dystrophy

Eteplirsen offers great hope to patients with Duchenne muscular dystrophy

Trace substance in sugar improves muscle regeneration in mouse model with muscular dystrophy

Trace substance in sugar improves muscle regeneration in mouse model with muscular dystrophy

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Cedars-Sinai team awarded grant to study cardiac stem cell treatment for Duchenne muscular dystrophy

Cedars-Sinai team awarded grant to study cardiac stem cell treatment for Duchenne muscular dystrophy

ARMGO Pharma gets $1 million to support ARM210 preclinical work for DMD treatment

ARMGO Pharma gets $1 million to support ARM210 preclinical work for DMD treatment

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

Study combines induced pluripotent stem cell technology and genetic correction to treat DMD

Study combines induced pluripotent stem cell technology and genetic correction to treat DMD