A genetic modifier of symptom onset in Pompe disease-Reference-Cited by-同舟云学术

A genetic modifier of symptom onset in Pompe disease

Published:2019-05 Issue: Volume:43 Page:553-561
ISSN:2352-3964
Container-title:EBioMedicine
language:en
Short-container-title:EBioMedicine

Author:

Bergsma Atze J.,in 't Groen Stijn L.M.,van den Dorpel Jan J.A.,van den Hout Hannerieke J.M.P.,van der Beek Nadine A.M.E.,Schoser Benedikt,Toscano Antonio,Musumeci Olimpia,Bembi Bruno,Dardis Andrea,Morrone Amelia,Tummolo Albina,Pasquini Elisabetta,van der Ploeg Ans T.,Pijnappel W.W.M. Pim^ORCID

Funder

Sophia Children's Hospital Foundation

Metakids

Publisher

Elsevier BV

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Reference37 articles.

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4. Long-term benefit of enzyme replacement therapy in Pompe disease: a 5-year prospective study;Kuperus;Neurology,2017

5. Pompe disease in Austria: clinical, genetic and epidemiological aspects;Loscher;J Neurol,2018

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