Childhood-onset striatonigral neurodegeneration robs children of the ability to walk and talk by age five. Researchers at ...

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...

This therapy comes in two steps: precision gene delivery, which uses short stretches ... The goal for releasing this groundbreaking research is to highlight how effective cell-specific therapies ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Mutations in BRCA2—a gene known to repair damaged DNA and suppress tumor formation—can predict an individual's predisposition ...

Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Mahidol University is undergoing a strategic transformation to strengthen its position as a world-class leader in health ...

LUXTURNA has significant market potential due to the high unmet need for effective treatments in rare genetic conditions. With its FDA approval for specific mutations in the RPE65 gene, it offers ...

Companies like Novartis say they are continuing gene therapy research. More than 95% of infants born in the U.S. with a rare neuromuscular disorder called spinal muscular atrophy are now treated ...