FIELD: medicine; pharmaceuticals.
SUBSTANCE: invention relates to a method for improving the pathological condition of the bone in a patient with acid sphingomyelinase deficiency (ASMD), and the method includes administering multiple doses of olipudase-alpha to the patient, where improvement in the abnormal condition of the bone is indicated by an improvement in the bone condition of the patient after multiple doses of olipudase-alpha.
EFFECT: technical result is alleviating the pathological conditions of the bone by enzyme replacement therapy using ASM in patients with ASMD, including an increase in their bone density and a decrease in their bone marrow "load".
23 cl, 4 dwg, 3 tbl, 1 ex
| Title |
Year |
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