Hemophilia

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Retrieved

2022-04-26

Source

Gard

Trials

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Genes

COX8A, F9, F8, TFPI, F2, ZAP70, AK3, PROC, F3, VWF, CCR5, F10, VIPR1, PROCR, TNF, F5, AAVS1, MTHFR, IFNA2, ADAMTS5, ABR, HAL, PRNP, PROS1, GH1, AMBP, IFNL3, CPB2, SLC4A1, HPSE2

COX8A, F9, F8, TFPI, F2, ZAP70, AK3, PROC, F3, VWF, CCR5, F10, VIPR1, PROCR, TNF, F5, AAVS1, MTHFR, IFNA2, ADAMTS5, ABR, HAL, PRNP, PROS1, GH1, AMBP, IFNL3, CPB2, SLC4A1, HPSE2, TAL1, C4orf3, ASPG, PRDX2, THBD, IL33, CARD14, UPF3B, KRT20, NT5C2, LAP, HAP1, ARTN, SCLY, CD163, ABCB6, SERPINA10, SOST, FOXP3, ST14, NHS, SELP, GPT, AGRP, APC, APOH, BPI, C4BPA, CALR, MS4A1, CLTC, CSF2, DMD, DPP4, F7, G6PD, HLA-A, CCL3L1, HLA-B, HNF4A, ICAM1, IL2RA, IL6, ISG20, KRT7, LTF, MRC1, NFYA, TNFRSF11B, SERPINA5, SERPINE2, C20orf181

Drugs

Adeno associated virus with modified transthyretin and sequence encoding factor IX variant gene, Adeno-associated viral vector containing the human factor IX gene, Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene

Adeno associated virus with modified transthyretin and sequence encoding factor IX variant gene, Adeno-associated viral vector containing the human factor IX gene, Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene, Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII, Albutrepenonacog alfa ( IDELVION ), Anti-inhibitor coagulant complex ( FEIBA ), Antihemophilic factor / Von Willebrand factor complex (human) ( ALPHANATE, HUMATE -P ), Autologous dendritic cells incubated ex vivo with zebularine and factor VIII, Desmopressine acetate ( DESMOPRESSIN ACETATE AVENTIS BEHRING L.L.C., OCTIM, MINIRIN ), Efmoroctocog alfa ( ELOCTA, ELOCTATE ), Eftrenonacog alfa ( ALPROLIX ), Factor VIII mimetic bispecific antibody, Fidanacogene elaparvovec, Fitusiran, Human coagulation factor IX (antihemophilic B factor) ( BETAFACT, ALPHANINE, NONAFACT ), Human coagulation factor VIII (antihemophilic A factor) ( FACTANE ), Human monoclonal IgG1 antibody against tissue factor pathway inhibitor, Human monoclonal IgG2 antibody against tissue factor pathway inhibitor, Humanised monoclonal IgG4 antibody against tissue factor pathway inhibitor, Humanised monoclonal modified IgG4 antibody with bispecific structure targeting factors IX, IXa, X and Xa ( HEMLIBRA ), Lentiviral vector encoding human coagulation factor IX, Long acting recombinantFactor VIIa-CTP3, Marzeptacog alfa (activated), Moroctocog alpha ( REFACTO AF ), Octocog alfa (liposomal), Octocog alpha ( ADVATE, HELIXATE NEXGEN, KOGENATE BAYER, KOVALTRY, IBLIAS ), Pegylated B-domain-deleted sequence-modified recombinant human factor VIII, Pegylated recombinant factor VIIa, Pegylated recombinant human factor IX ( REFIXIA ), Recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA, Recombinant adeno-associated viral vector serotype 6 encoding the B-domain-deleted human factor VIII, Recombinant coagulation factor VIIa (rFVIIa) ( NOVOSEVEN, NOVOSEVEN RT ), Recombinant factor VIIa modified with three terminal repeats derived from the beta chain of human chorionic gonadotropin, Recombinant fusion protein linking human coagulation factor VIIa with human albumin (rVIIa-FP), Recombinant porcine factor VIII (B domain deleted) ( OBIZUR ), Sequence-modified human recombinant factor VIIa, Turoctocog alfa pegol ( ESPEROCT ), Vatreptacog alfa (activated), nonacog alfa ( BENEFIX ), nonacog gamma ( RIXUBIS ), recombinant AAV vector with hu37 serotype capsid (AAVhu37) encoding human FVIII, recombinant adeno-associated viral vector containing a bioengineered capsid and a codon-optimised expression cassette to drive the expression of the SQ form of a B-domain deleted human coagulation factor VIII, recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant, recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein, recombinant human factor IX protein modified with three point mutations

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Hemophilia is a bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding following an injury, surgery, or having a tooth pulled. In severe cases, heavy bleeding occurs after minor trauma or in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. The major types of this disorder are hemophilia A and hemophilia B. Although the two types have very similar signs and symptoms, they are caused by mutations in different genes. People with an unusual form of hemophilia B, known as hemophilia B Leyden, experience episodes of excessive bleeding in childhood, but have few bleeding problems after puberty. Another form of the disorder, acquired hemophilia, is not caused by inherited gene mutations.[4874]