Enzyme replacement therapy interruption in mucopolysaccharidosis type IVA patients and its impact in different clinical outcomes-Reference-Cited by-同舟云学术

Enzyme replacement therapy interruption in mucopolysaccharidosis type IVA patients and its impact in different clinical outcomes

Published:2021-01-12 Issue:1 Volume:58 Page:104-113
ISSN:2192-8312
Container-title:JIMD Reports
language:en
Short-container-title:JIMD Reports

Author:

Politei Juan¹,Porras‐Hurtado Gloria Liliana²,Guelbert Norberto³,Fainboim Alejandro⁴,Horovitz Dafne Dain Gandelman⁵,Satizábal José María⁶

Affiliation:

1. Laboratorio de Neuroquímica Dr. N. A. Chamoles Fundación para el Estudio de Enfermedades Neurometabólicas (FESEN) Buenos Aires Argentina

2. Clinica Comfamiliar Risaralda Colombia

3. Hospital de Niños de la Santísima Trinidad, Enfermedades Metabolicas CEMECO Córdoba Argentina

4. Hospital de Niños Ricardo Gutiérrez Buenos Aires Argentina

5. Departamento de Genética Médica Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira/Fiocruz Rio de Janeiro Brazil

6. Departamento Ciencias Fisiológicas Escuela de Ciencias Básicas, Grupo de investigación Enfermedades Congénitas del Metabolismo, Facultad de Salud, Universidad del Valle Cali Colombia

Publisher

Wiley

Subject

Biochemistry, Genetics and Molecular Biology (miscellaneous),Endocrinology, Diabetes and Metabolism,Internal Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jmd2.12192

Reference27 articles.

1. International Morquio A Registry: Clinical manifestation and natural course of Morquio A disease

2. Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management

3. Respiratory and sleep disorders in mucopolysaccharidosis

4. Heart and Cardiovascular Involvement in Patients with Mucopolysaccharidosis Type IVA (Morquio-A Syndrome)

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