Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study-Reference-Cited by-同舟云学术

Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study

Published:2021-05 Issue:1 Volume:133 Page:94-99
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Horovitz Dafne D.G.,Leão Emília K.E.A.,Ribeiro Erlane M.,Martins Ana M.,Barth Anneliese L.,Neri João I.C.F.,Kerstenetzky Marcelo,Siqueira Ana C.M.,Ribeiro Bethania F.R.,Kim Chong A.,Santos Francisca C.,Franco José F.S.,Lichtvan Leniza C.L.,Giuliani Liane R.,Rodrigues Maria Do C.S.,Bonatti Renata C.F.,Teixeira Thaís B.,Gonçalves Alexandra,Lourenço Charles M.,Pereira Ane S.S.,Acosta Angelina X.

Publisher

Elsevier BV

Subject

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

Reference19 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Clinical and biochemical study of 28 patients with mucopolysaccharidosis type VI;Azevedo;Clin Genet.,2004

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. Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future;Chen;J. Hum. Genet.,2019

5. Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age–a sibling control study;McGill;Clin. Genet.,2010

Cited by 9 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

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. Causes of death in mucopolysaccharidoses;Molecular Genetics and Metabolism;2024-07

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

4. Metabolic Cardiomyopathies and Cardiac Defects in Inherited Disorders of Carbohydrate Metabolism: A Systematic Review;International Journal of Molecular Sciences;2023-05-11

5. Early enzyme replacement therapy prevents dental and craniofacial abnormalities in a mouse model of mucopolysaccharidosis type VI;Frontiers in Physiology;2022-09-21