FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology and represents a method of treating acid sphingomyelinase (asmd) deficiency in a human subject, involving administering recombinant human acid sphingomyelinase (rhasm) to a human subject in a dose-increasing regimen.
EFFECT: invention provides higher clinical effectiveness in acid sphingomyelinase deficiency.
30 cl, 7 dwg, 9 tbl, 4 ex
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Authors
Dates
2025-01-28—Published
2020-09-02—Filed