Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

Pompe disease is an inherited neuromuscular disease caused by deficiency of lysosomal acid alpha-glucosidase (GAA) leading to glycogen accumulation in muscle and motoneurons. Cardiopulmonary failure in infancy leads to early mortality, and GAA enzyme replacement therapy (ERT) results in improved sur...

Descrición completa

Gardado en:
Detalles Bibliográficos
Main Authors: Smith, Barbara K., Collins, Shelley W., Conlon, Thomas J., Mah, Cathryn S., Lawson, Lee Ann, Martin, Anatole D., Fuller, David D., Cleaver, Brian D., Clément, Nathalie, Phillips, Dawn, Islam, Saleem, Dobjia, Nicole, Byrne, Barry J.
Formato: Artigo
Idioma:Inglês
Publicado: Mary Ann Liebert, Inc. 2013
Assuntos:
Research Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3689178/
https://ncbi.nlm.nih.gov/pubmed/23570273
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.250
Tags: Engadir etiqueta
Sen Etiquetas, Sexa o primeiro en etiquetar este rexistro!

Títulos similares