ENZYME REPLACEMENT THERAPY WITH DOSE ESCALATION FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY Russian patent published in 2025 - IPC A61K38/43 

Abstract RU 2833674 C2

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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RU 2 833 674 C2

Authors

Schuchmann, Edward, H.

Desnick, Robert, J.

Cox, Gerald, F.

Andrews, Laura, P.

Murray, James, M.

Dates

2025-01-28Published

2020-09-02Filed