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Mevalonate Kinase Deficiency

Inborn Errors Working Party (IEWP)
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Primary objective:
In this retrospective study we aim to evaluate the efficacy and safety of stem cell transplantation in MKD
Key inclusion criteria:
All MKD patients who had undergone allogeneic stem cell transplantation
Principal investigator:
Joost Frenkel, Jerold Jeyaratman and Arjan Lankester
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EBMT Study coordinator:
Tiar Sirait
Study coordinator email: