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...

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Autores principales: 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
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2013
Materias:
Research Articles
Acceso en línea: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
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