Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa-Reference-Cited by-同舟云学术

Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa

Published:2022-09 Issue:1-2 Volume:137 Page:164-172
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Mitchell John J.,Burton Barbara K.,Bober Michael B.,Campeau Philippe M.,Cohen Shelda,Dosenovic Sara,Ellaway Carolyn,Bhattacharya Kaustuv,Guffon Nathalie,Hinds David,Lail Alice,Lin Shuan-Pei,Magner Martin,Raiman Julian,Schwartz-Sagi Liat,Stepien Karolina M.

Funder

McGill University

BioMarin Pharmaceutical

Publisher

Elsevier BV

Subject

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

Reference19 articles.

1. Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA;Hendriksz;Mol. Genet. Metab.,2013

2. International Morquio A registry: clinical manifestation and natural course of Morquio A disease;Montaño;J. Inherit. Metab. Dis.,2007

3. The Morquio A clinical assessment program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects;Harmatz;Mol. Genet. Metab.,2013

4. Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA;Hendriksz;J. Inherit. Metab. Dis.,2012

5. Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues;Hendriksz;Orphan. J. Rare Dis.,2016

Cited by 1 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. Follow-up patterns and Multidisciplinary Clinical Approaches in Patients with Mucopolysaccharidosis Type IVA in Colombia;Pediatría;2024-03-30