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

• প্রধান লেখক: Jerold Jeyaratnam (লেখক), Maura Faraci (লেখক), Andrew R. Gennery (লেখক), Katarzyna Drabko (লেখক), Mattia Algeri (লেখক), Akira Morimoto (লেখক), Tiarlan Sirait (লেখক), Arjan C. Lankester (লেখক), Michael Albert (লেখক), Benedicte Neven (লেখক), Joost Frenkel (লেখক), on behalf of the EBMT Inborn Errors Working Party (লেখক)
• বিন্যাস: গ্রন্থ
• প্রকাশিত: BMC, 2022-07-01T00:00:00Z.
• বিষয়গুলি: article
• অনলাইন ব্যবহার করুন: Connect to this object online.

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...