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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...

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Kaydedildi:
Detaylı Bibliyografya
Yayımlandı:Mol Genet Metab
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2017
Konular:
Online Erişim: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
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