Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry

Autor:	Joel Charrow, Manisha Balwani, Hans C. Andersson, Thomas A. Burrow, Barry E. Rosenbloom, Neal J. Weinreb, Edwin H. Kolodny, Aneal Khan, Priya S. Kishnani, Julie L. Batista, C. Ronald Scott, Pramod K. Mistry, Paige Kaplan
Rok vydání:	2017
Předmět:	Adult Male 0301 basic medicine congenital hereditary and neonatal diseases and abnormalities Pediatrics medicine.medical_specialty Adolescent Imiglucerase medicine.medical_treatment Splenectomy Disease 03 medical and health sciences 0302 clinical medicine Alglucerase Prevalence medicine Humans Enzyme Replacement Therapy Registries Research Articles Gaucher Disease business.industry nutritional and metabolic diseases Hematology Enzyme replacement therapy Middle Aged Glucosylceramidase Surgery Clinical trial 030104 developmental biology 030220 oncology & carcinogenesis Lower prevalence Female business Follow-Up Studies Research Article medicine.drug
Zdroj:	American Journal of Hematology
ISSN:	0361-8609
Popis:	This study tests the hypothesis that the prevalence of severe clinical manifestations in Gaucher disease type 1 (GD1) patients at the time of treatment initiation has changed since alglucerase/imiglucerase enzyme replacement therapy (ERT) was approved in the United States (US) in 1991. US alglucerase/imiglucerase‐treated GD1 patients from the International Collaborative Gaucher Group Gaucher Registry clinicaltrials.gov NCT00358943 were stratified by age at ERT initiation (
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c3cf4ef54b043861ad98aba02a023072 https://doi.org/10.1002/ajh.24801 Zobrazit plný text záznamu Plný text