Overall, the long-term follow-up of HSCT in MPS IVA patients showed that this therapy achieved the donor’s GALNS activity level, improved pulmonary function, BMD and ADL, and reduced the frequency of surgical intervention, suggesting that HSCT could be a useful supportive treatment option for patients with MPS IVA [10, 30, 32]. Here, GALNS is linked to mucopolysaccharidosis type 4A.