Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease

PURPOSE: To characterize clinical characteristics and genotypes of patients in the ADVANCE study of 4000 L-scale alglucosidase alfa (NCT01526785), the largest prospective United States Pompe disease cohort to date. METHODS: Patients aged ≥1 year with confirmed Pompe disease previously receiving 160 ...

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Detalles Bibliográficos
Autores principales: Kishnani, Priya S., Gibson, James B., Gambello, Michael J., Hillman, Richard, Stockton, David W., Kronn, David, Leslie, Nancy D., Pena, Loren D. M., Tanpaiboon, Pranoot, Day, John W., Wang, Raymond Y., Goldstein, Jennifer L., An Haack, Kristina, Sparks, Susan E., Zhao, Yang, Hahn, Si Houn
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8076035/
https://www.ncbi.nlm.nih.gov/pubmed/31086307
http://dx.doi.org/10.1038/s41436-019-0527-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8076035/
https://www.ncbi.nlm.nih.gov/pubmed/31086307
http://dx.doi.org/10.1038/s41436-019-0527-9

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