12-year-old with ultra-rare genetic disorder recovers after life-saving transplant
Doctors have successfully treated India’s first known case of MYSM1-related bone marrow failure syndrome (BMFS4), an extremely rare genetic condition, using a half-matched (haploidentical) bone marrow transplant.MYSM1-related bone marrow failure is an extremely uncommon hereditary condition resulting from mutations in both alleles of the MYSM1 gene. There have been fewer than 20 cases reported around…