FIELD: medicine; therapy.
SUBSTANCE: group of inventions relates to the field of medicine, namely to therapy; it is intended for the treatment of GCase path disfunction (glucocerebrosidase enzyme). A composition for the treatment of GCase path disfunction contains glucocerebrosidase (hereinafter – GCB) and isofagomine (IFM) at a molar ratio of at least about 1:2.5. The composition is intended for subcutaneous injection. In other embodiments, a method for the production of the specified composition, methods for the treatment of GCase path disfunction, and the use of the specified composition in the production of a drug, as well as a container containing the specified composition are presented.
EFFECT: use of the group of inventions allows for an increase in the activity and bioavailability, which reduces a number of subcutaneous injections, which are necessary to be injected to a subject, as well as provides improved stability allowing for maintenance of high concentrations of circulating GCB, which leads to greater absorption of GCB by monocytes, macrophages, and tissue histiocytes.
73 cl, 21 dwg, 13 tbl, 19 ex
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Authors
Dates
2023-02-15—Published
2018-10-25—Filed