Juvenile Myelomonocytic Leukemia (JMML) is a rare and aggressive blood cancer mainly affecting young children. It is characterized by the presence of somatic PTPN11 mutations in approximately 35% of cases. Current treatments, like chemotherapy, are not very effective, and stem cell transplants are the only potential cure, but they only work for about half of patients, with relapse being the leading cause of death. Surprisingly, there are currently no FDA-approved medications for treating JMML. Chronic inflammation seems to be involved in JMML development.
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