P141 Mirage syndrome with bone marrow stem cell transplant: A case report

MIRAGE (myelodysplasia, infection, restriction of growth, adrenal hypoplasia, genital phenotypes and enteropathy) syndrome is a recently described multisystem disorder that is related to gain of function of the growth repressor SAMD9. Due to the dominant suppressive effect of SAMD9 mutants, MIRAGE syndrome is associated with high mortality. A new publication this year suggests bone marrow transplantation (BMT) may be necessary for survival. Here...