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

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...

Source: Pediatric Rheumatology - July 29, 2022 Category: Rheumatology Authors: Jerold Jeyaratnam, Maura Faraci, Andrew R. Gennery, Katarzyna Drabko, Mattia Algeri, Akira Morimoto, Tiarlan Sirait, Arjan C. Lankester, Michael Albert, Benedicte Neven and Joost Frenkel Tags: Research article Source Type: research