In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
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