Enzyme Replacement Therapy with Elosulfase alfa for Mucopolysaccharidosis IVA (Morquio A Syndrome): Milestones and Challenges-Reference-Cited by-同舟云学术

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Enzyme Replacement Therapy with Elosulfase alfa for Mucopolysaccharidosis IVA (Morquio A Syndrome): Milestones and Challenges

Published:2017-08-25 Issue:9 Volume:5 Page:741-752
ISSN:2167-8707
Container-title:Expert Opinion on Orphan Drugs
language:en
Short-container-title:Expert Opinion on Orphan Drugs

Author:

Puckett Yana¹,Mulinder Holly¹,Montaño Adriana M.²³

Affiliation:

1. Department of General Surgery, Texas Tech University, School of Medicine, Lubbock, TX, USA

2. Department of Pediatrics, Saint Louis University, School of Medicine, Edward A. Doisy Research Center, St. Louis, MO, USA

3. Department of Biochemistry and Molecular Biology, Saint Louis University, School of Medicine, Edward A. Doisy Research Center, St. Louis, MO, USA

Publisher

Informa UK Limited

Subject

Pharmacology (medical),Health Policy,Pharmacology, Toxicology and Pharmaceutics (miscellaneous)

Link

https://www.tandfonline.com/doi/pdf/10.1080/21678707.2017.1366900

Reference67 articles.

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

2. Morquio A Syndrome‐Associated Mutations: A Review of Alterations in the GALNS Gene and a New Locus‐Specific Database

3. Mucopolysaccharidosis IVA: identification of mutations and methylation study in GALNS gene

4. Four novel mutations in the N-acetylgalactosamine-6-sulfate sulfatase gene among Egyptian patients with Morquio A disease

5. GALNSmutations in Indian patients with mucopolysaccharidosis IVA

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1. Novel human recombinant N-acetylgalactosamine-6-sulfate sulfatase produced in a glyco-engineered Escherichia coli strain;Heliyon;2024-06

2. Hydrogel Delivery Device for the In Vitro and In Vivo Sustained Release of Active rhGALNS Enzyme;Pharmaceuticals;2023-06-27

3. Mucopolysaccharidosis Type IVA: Extracellular Matrix Biomarkers in Cardiovascular Disease;Frontiers in Cardiovascular Medicine;2022-05-10

4. Umbilical mesenchymal stem cell-derived extracellular vesicles as enzyme delivery vehicle to treat Morquio A fibroblasts;Stem Cell Research & Therapy;2021-05-06

5. Biodegradable polyethylene glycol hydrogels for sustained release and enhanced stability of rhGALNS enzyme;Drug Delivery and Translational Research;2020-01-28

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