Comprehensive and long‐term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3-Reference-Cited by-同舟云学术

Comprehensive and long‐term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3

Published:2022-12-23 Issue:3 Volume:70 Page:
ISSN:1545-5009
Container-title:Pediatric Blood & Cancer
language:en
Short-container-title:Pediatric Blood & Cancer

Author:

Anurathapan Usanarat¹,Tim‐Aroon Thipwimol²,Zhang Wujuan³⁴,Sanpote Watinee⁵,Wongrungsri Siranee⁵,Khunin Nitcha²,Chutipongtanate Somchai⁶,Chirdkiatgumchai Vilawan⁷,Ngiwsara Lukana⁸,Jaovisidha Suphaneewan⁹,Khongkraparn Arthaporn²,Pakakasama Samart¹^ORCID,Svasti Jisnuson⁸^ORCID,Setchell Kenneth D. R.³⁴,Wattanasirichaigoon Duangrurdee²^ORCID,Hongeng Suradej¹^ORCID

Affiliation:

1. Division of Hematology and Oncology Department of Pediatrics Faculty of Medicine Ramathibodi Hospital Mahidol University Bangkok Thailand

2. Division of Medical Genetics Department of Pediatrics Faculty of Medicine Ramathibodi Hospital Mahidol University Bangkok Thailand

3. Department of Pathology and Laboratory Medicine Cincinnati Children's Hospital Medical Center Cincinnati Ohio USA

4. Department of Pediatrics University of Cincinnati College of Medicine Cinicnnati Ohio USA

5. Department of Pediatrics Lampang Hospital Lampang Thailand

6. Department of Environmental and Public Health Sciences University of Cincinnati College of Medicine Cincinnati Ohio USA

7. Division of Developmental and Behavioral Pediatrics Department of Pediatrics Faculty of Medicine Ramathibodi Hospital Mahidol University Bangkok Thailand

8. Laboratory of Biochemistry Chulabhorn Research Institute Bangkok Thailand

9. Department of Diagnostic and Therapeutic Radiology Faculty of Medicine Ramathibodi Hospital Mahidol University Bangkok Thailand

Publisher

Wiley

Subject

Oncology,Hematology,Pediatrics, Perinatology and Child Health

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/pbc.30149

Reference36 articles.

1. Glucosylsphingosine is a key biomarker of Gaucher disease

2. Multi-system disorders of glycosphingolipid and ganglioside metabolism

3. The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?

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1. Hematopoietic stem cell transplantation or enzyme replacement therapy in Gaucher disease type 3;Molecular Genetics and Metabolism;2024-08

2. Noninvasive DBS-Based Approaches to Assist Clinical Diagnosis and Treatment Monitoring of Gaucher Disease;Biomedicines;2023-09-29