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.
Research on dystrophin gene sequence could lead to treatments for Duchenne muscular dystrophy

Research on dystrophin gene sequence could lead to treatments for Duchenne muscular dystrophy

Potential stem cell therapy for Duchenne’s

Potential stem cell therapy for Duchenne’s

Positive gene therapy results in large mammals of Duchenne muscular dystrophy

Positive gene therapy results in large mammals of Duchenne muscular dystrophy

Stem cell transplantation prevents decrease in heart function associated with DMD

Stem cell transplantation prevents decrease in heart function associated with DMD

The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies

The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies

Dantrolene shows promise for treating DMD

Dantrolene shows promise for treating DMD

Dantrolene may help combat Duchenne muscular dystrophy in boys

Dantrolene may help combat Duchenne muscular dystrophy in boys

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

‘Weekend pill’ employed for muscular dystrophy

‘Weekend pill’ employed for muscular dystrophy

Tadalafil may offer therapeutic strategy in patients with Becker muscular dystrophy

Tadalafil may offer therapeutic strategy in patients with Becker muscular dystrophy

HCT 1026 may help stem debilitating effects of muscular dystrophy

HCT 1026 may help stem debilitating effects of muscular dystrophy

Microtubule stiffening worsens skeletal muscle degeneration in Duchenne muscular dystrophy

Microtubule stiffening worsens skeletal muscle degeneration in Duchenne muscular dystrophy

Laminin-111 protein therapy could treat Duchenne muscular dystrophy

Laminin-111 protein therapy could treat Duchenne muscular dystrophy

Read-through molecule improves muscle function in DMD

Read-through molecule improves muscle function in DMD

New compound could treat certain types of genetic disorders in muscles

New compound could treat certain types of genetic disorders in muscles

TACT believes that Halo's HT-100 may effectively treat DMD

TACT believes that Halo's HT-100 may effectively treat DMD

Prosensa secures €23M in new equity financing

Prosensa secures €23M in new equity financing

New gene therapy approach safe for patients with muscular dystrophy

New gene therapy approach safe for patients with muscular dystrophy

Critical breakthrough in development of treatment for Duchenne Muscular Dystrophy

Critical breakthrough in development of treatment for Duchenne Muscular Dystrophy

PTC restructures collaboration with Genzyme

PTC restructures collaboration with Genzyme