2 Minute Medicine5 天
AAV9.LAMP2B gene therapy shows clinical benefit in Danon disease
In seven patients with Danon disease, a single infusion of adeno-associated virus serotype 9 carrying transgene LAMP2B ...
pharmaphorum3 天
Rocket Pharmaceuticals in orbit after gene therapy read-out
Danon Disease is a rare X-linked disorder caused by genetic mutations in the LAMP2 gene and the therapy works by instructing the body to express a healthy copy of the LAMP2B protein in order to ...