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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Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2013
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Research Articles
On-line přístup: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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