Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...

NS Pharma, Inc. (NS Pharma), a biopharmaceutical leader in rare disease and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the National Center of Neurology and ...

People with Duchenne muscular dystrophy carry a mutation in the DMD gene coding for dystrophin, a protein crucial for proper muscle function. Scientists are working to treat the disease at its genetic ...

Which do you think would win in a fight between gene editing and gene therapy? Well, we might find out sooner than expected, because Vertex Pharmaceuticals (NASDAQ: VRTX) has doubled down on a ...

Duchenne muscular dystrophy is caused by mutations in the dystrophin encoding DMD gene that disrupt the reading frame. Muscle fibers lacking dystrophin are more sensitive to damage and over time ...

Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows. The ...

Shares of Sarepta Therapeutics rose nearly 5% in early premarket trading on Wednesday as the company prepared to present new ...

The powerful gene editing technology CRISPR is one small step closer to treating a human disease. In a new paper published in Science, researchers led by Eric Olson, professor and chair of molecular ...

Vertex Pharmaceuticals recently doubled down on its gene editing collaboration with CRISPR Therapeutics, but it's miles behind an arguably simpler solution. DMD is a rare inherited muscle-wasting ...