Disease

Bone marrow failure syndrome 41

Gene

MYSM1

Inheritance AR
Current Treatments Hematopoietic stem cell transplantation (HSCT) - bone marrow transplant
Evidence for Treatment expert opinion
Non-Molecular Testing Availability yes
Non-Molecular Test WBC and RBC counts. immunoglobulin level, T and B Lymphocyte and Natural Killer Cell Profile2
Experimental / Investigational Therapies https://clinicaltrials.gov/ct2/results?cond=Bone+Marrow+Failure+Syndrome&term=&cntry=&state=&city=&dist=
Last Updated: March 5, 2026
RX Genes
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