ENZYME REPLACEMENT THERAPY WITH DOSE INCREASE FOR TREATMENT OF ACID SPHINGOMYELINASE DEFICIENCY Russian patent published in 2015 - IPC A61K38/43 

FIELD: medicine.

SUBSTANCE: group of inventions relates to medicine and is intended for treatment of human subjects with acid sphingomyelinase deficiency. Treatment is realised with introduction of initial low non-toxic dose of acid sphingomyelinase (ASM). After that, high ASM doses are introduced successively. Monitoring with respect of one or several undesirable side effects is carried out after each following dose, as indicators of which used are: increased concentration of total bilirubin, concentration of ceramide in plasma, concentration of C-reactive protein (CRP) in plasma or associated undesirable phenomenon. After that, supporting scheme, including introduction of ASM dose, equal or lower than the highest dose, tolerated by subject, to subject as supporting dose, is applied. In other version of invention treatment includes introduction of acid sphingomyelinase (ASM) by scheme with dose increase in the following successive doses: 0.1 mg/kg, 0.3 mg/kg and 0.6 mg/kg.

EFFECT: application of group of inventions ensures safe introduction of high dose of ASM enzyme, which is accompanied by fewer side effects.

35 cl, 15 dwg, 9 tbl, 4 ex