Long-term evolution of mucopolysaccharidosis type I in twins treated with enzyme replacement therapy plus hematopoietic stem cells transplantation

Long-term evolution of mucopolysaccharidosis type I in twins treated with enzyme replacement therapy plus hematopoietic stem cells transplantation

Mucopolysaccharidoses (MPSs) are a heterogeneous group of diseases that have in common the accumulation of glycosaminoglycans (mucopolysaccharides) within the lysosome. The diseases are caused by a deficiency of the enzyme α-L-iduronidase which is responsible for the degradation of glycosaminoglycan...

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Bibliographic Details
Main Authors: Luis M. Carbajal-Rodríguez, Martín Pérez-García, Raymundo Rodríguez-Herrera, Haydeé Salazar Rosales, Alberto Olaya-Vargas
Format: Article
Language:English
Published: Elsevier 2021-08-01
Series:Heliyon
Subjects:
Enzyme replacement therapy
Hematopoietic stem cell transplantation
Monochorionic diamniotic twins
Mucopolysaccharidosis type I
Pediatrics
Online Access:http://www.sciencedirect.com/science/article/pii/S2405844021018430

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http://www.sciencedirect.com/science/article/pii/S2405844021018430

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