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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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Uloženo v:
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: BMC 2022-07-01
Edice:Pediatric Rheumatology Online Journal
Témata:
Inborn metabolic disease
Mevalonic aciduria
Therapeutic options
Auto-inflammation
Allogeneic stem cell transplantation
On-line přístup:https://doi.org/10.1186/s12969-022-00716-4
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