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Clinical, biochemical and genetic profiles of patients with mucopolysaccharidosis type IVA (Morquio A syndrome) in Malaysia: the first national natural history cohort study

Crossref DOI link: https://doi.org/10.1186/s13023-019-1105-6

Published Online: 2019-06-14

Published Print: 2019-12

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Leong, Huey Yin https://orcid.org/0000-0002-0294-6823
Abdul Azize, Nor Azimah

Chew, Hui Bein

Keng, Wee Teik

Thong, Meow Keong

Mohd Khalid, Mohd Khairul Nizam

Hung, Liang Choo

Mohamed Zainudin, Norzila

Ramlee, Azura

Md Haniffa, Muzhirah Aisha

Yakob, Yusnita

Ngu, Lock Hock
Funding

Funding for this research was provided by:

BioMarin Pharmaceutical
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Article History

Received: 16 March 2019

Accepted: 26 May 2019

First Online: 14 June 2019

Ethics approval and consent to participate

: Informed consent to participate in this study was obtained from study participants and their parents. This study was approved by the Medical Research and Ethics Committee, Ministry of Health, Malaysia.

: Informed consent was obtained from all study participants and/or their parents for the purpose of this publication.

: HYL, LHN, HBC and MKT have received honoraria from BioMarin and Genzyme for work outside of this study. HLC has received honoraria from BioMarin.

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