Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease

Pompe disease is a lysosomal storage disorder caused by acid-α-glucosidase (GAA) deficiency, leading to glycogen storage. The disease manifests as a fatal cardiomyopathy in infantile form. Enzyme replacement t...

from #ORL-AlexandrosSfakianakis via ola Kala on Inoreader http://ift.tt/2xNzgJ2