IFitusiran

Fitusiran (API)

Isicelo soPhando:
I-Fitusiran API yi-RNA encinci ephazamisayo (i-siRNA) ephandwe ngokukodwa kwintsimi ye-hemophilia kunye nokuphazamiseka kwe-coagulation. Ijolise kwii-antithrombin (AT okanye i-SERPINC1)gene esibindini ukunciphisa imveliso ye-antithrombin. Abaphandi basebenzisa i-Fitusiran ukuphonononga iindlela zokuphazamiseka kwe-RNA (i-RNAi), ukuthuliswa kofuzo oluthile lwesibindi, kunye nezicwangciso zonyango ezinoveli zokubuyisela i-coagulation kwi-hemophilia A kunye ne-B izigulane, kunye okanye ngaphandle kwe-inhibitors.

Umsebenzi:
I-Fitusiran isebenza ngokuthulisa ukubonakaliswa kwe-antithrombin, i-anticoagulant yendalo, ngaloo ndlela ikhulisa ukuveliswa kwe-thrombin kunye nokukhuthaza ukubunjwa kwehlwili. Lo matshini unikezela ngendlela yonyango lweprophylactic yokunciphisa iziqendu ezophayo kwizigulane ezine-hemophilia. Njenge-API, i-Fitusiran isebenza njengesithako esisebenzayo kwi-long-acting subcutaneous therapies ejoliswe ekuphuculeni umgangatho wobomi kunye nokunciphisa umthwalo wonyango ekuphazamisekeni kwegazi.