The efficacy and safety of allogeneic stem cell transplantation in Mevalonate Kinase Deficiency

Abstract Objectives Mevalonate kinase deficiency (MKD) is a rare autoinflammatory syndrome. Several reports have described allogeneic hematopoietic stem cell transplantation in severely affected patients, sometimes with promising results. In view of the scarcity of data, this study aims to analyse t...

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發表在:	Pediatric Rheumatology Online Journal
Main Authors:	Jerold Jeyaratnam, Maura Faraci, Andrew R. Gennery, Katarzyna Drabko, Mattia Algeri, Akira Morimoto, Tiarlan Sirait, Arjan C. Lankester, Michael Albert, Benedicte Neven, Joost Frenkel, on behalf of the EBMT Inborn Errors Working Party
格式:	Article
語言:	英语
出版:	BMC 2022-07-01
主題:	Inborn metabolic disease Mevalonic aciduria Therapeutic options Auto-inflammation Allogeneic stem cell transplantation
在線閱讀:	https://doi.org/10.1186/s12969-022-00716-4