Early Treatment with Alglucosidase Alfa Prolongs Long Term Survival of Infants with Pompe Disease

In a previous 52-week trial, treatment with alglucosidase alfa markedly improved cardiomyopathy, ventilatory function, and overall survival among 18 children <7 months old with infantile-onset Pompe disease. Sixteen of the 18 patients enrolled in an extension study, where they continued to receiv...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Main Authors: Kishnani, Priya S., Corzo, Deya, Leslie, Nancy D., Gruskin, Daniel, van der Ploeg, Ans, Clancy, John P., Parini, Rosella, Morin, Gilles, Beck, Michael, Bauer, Mislen S., Jokic, Mikael, Tsai, Chen-En, Tsai, Brian W.H., Morgan, Claire, O’Meara, Tara, Richards, Susan, Tsao, Elisa C., Mandel, Hanna
Formato: Artigo
Idioma:Inglês
Publicado em: 2009
Assuntos:
Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3129995/
https://ncbi.nlm.nih.gov/pubmed/19542901
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1203/PDR.0b013e3181b24e94
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados