I-Fitusiran

I-Fitusiran (API)

Isicelo socwaningo:
I-Fitusiran API iyi-RNA encane ephazamisayo (siRNA) ephenywa ngokuyinhloko emkhakheni we-hemophilia kanye nokuphazamiseka kwe-coagulation. Ihlose ii-antithrombin (AT noma i-SERPINC1)isakhi sofuzo esibindini ukunciphisa ukukhiqizwa kwe-antithrombin. Abacwaningi basebenzisa i-Fitusiran ukuhlola izindlela zokuphazanyiswa kwe-RNA (i-RNAi), ukuthulisa izakhi zofuzo ezithize esibindini, kanye namasu anoveli okwelapha okubuyisela ukulinganisa ukuhlangana ezigulini ze-hemophilia A no-B, ezinama-inhibitors noma ngaphandle kwazo.

Umsebenzi:
I-Fitusiran isebenza ngokuthulisa ukuvezwa kwe-antithrombin, i-anticoagulant yemvelo, ngaleyo ndlela ikhulise ukukhiqizwa kwama-thrombin futhi ikhuthaze ukwakheka kwamahlule. Lo mshini unikeza indlela yokwelapha ye-prophylactic yokunciphisa iziqephu zokopha ezigulini ezine-hemophilia. Njenge-API, i-Fitusiran isebenza njengesithako esisebenzayo ekwelashweni okuhlala isikhathi eside okungaphansi kwesikhumba okuhloswe ngayo ukuthuthukisa izinga lempilo kanye nokunciphisa umthwalo wokwelashwa ezinkingeni zokopha.