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Morquio A syndrome and effect of enzyme replacement therapy in different age groups of Turkish patients: a case series

BACKGROUND: This case series includes longitudinal clinical data of ten patients with Morquio A syndrome from south and southeastern parts of Turkey, which were retrospectively collected from medical records. All patients received enzyme replacement therapy (ERT). Clinical data collected included ph...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Kılavuz, Sebile, Basaran, Sibel, Kor, Deniz, Bulut, Fatma Derya, Erdem, Sevcan, Ballı, Hüseyin Tuğsan, Dağkıran, Muhammed, Bisgin, Atil, Mungan, Halise Neslihan Önenli
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2021
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7983100/
https://ncbi.nlm.nih.gov/pubmed/33752727
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01761-0
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