The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)-Reference-Cited by-同舟云学术

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The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

Published:2017-09 Issue:1-2 Volume:122 Page:107-112
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Harmatz P.,Hendriksz C.J.,Lampe C.,McGill J.J.,Parini R.,Leão-Teles E.,Valayannopoulos V.,Cole T.J.,Matousek R.,Graham S.,Guffon N.,Quartel A.

Funder

BioMarin Pharmaceutical Inc.

National Institutes of Health (NIH)

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference32 articles.

1. Mucopolysaccharidosis VI;Valayannopoulos;Orphanet J. Rare Dis.,2010

2. Clinical characteristics of adults with slowly progressing mucopolysaccharidosis VI: a case series;Thümler;J. Inherit. Metab. Dis.,2012

3. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome);Swiedler;Am. J. Med. Genet. A,2005

4. Growth charts for individuals with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome);Quartel;JIMD Rep.,2015

5. Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study

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1. Real-world pharmacovigilance analysis of galsulfase: a study based on the FDA adverse event reporting system (FAERS) database;Frontiers in Pharmacology;2024-08-05

2. Pain management challenges in a patient with mucopolysaccharidosis IVA;Clinical Case Reports;2024-07-28

3. Consensus-based expert recommendations on the management of MPS IVa and VI in Saudi Arabia;Orphanet Journal of Rare Diseases;2024-07-17

4. Elucidating the functional impact of G137V and G144R variants in Maroteaux Lamy’s Syndrome by Molecular Dynamics Simulation;Molecular Diversity;2023-07-17

5. Persistent bone and joint disease despite current treatments for mucopolysaccharidosis types I, II, and VI: Data from a 10‐year prospective study;Journal of Inherited Metabolic Disease;2023-03-06

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