Carregant...

Insight into the phenotype of infants with Pompe disease identified by newborn screening with the common c.-32-13T>G “late-onset” GAA variant

OBJECTIVE: Newborn screening (NBS) has led to early diagnosis and early initiation of treatment for infantile onset Pompe Disease (IOPD). However, guidelines for management of late onset Pompe disease (LOPD) via NBS, especially with the IVS c.-32-13T>G are not clear. This IVS variant is noted in...

Descripció completa

Guardat en:
Dades bibliogràfiques
Publicat a:Mol Genet Metab
Autors principals: Rairikar, Mugdha V., Case, Laura E., Bailey, Lauren A., Kazi, Zoheb B., Desai, Ankit K., Berrier, Kathryn L., Coats, Julie, Gandy, Rachel, Quinones, Rebecca, Kishnani, Priya S.
Format: Artigo
Idioma:Inglês
Publicat: 2017
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5722675/
https://ncbi.nlm.nih.gov/pubmed/28951071
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.09.008
Etiquetes: Afegir etiqueta
Sense etiquetes, Sigues el primer a etiquetar aquest registre!