Hemophilia
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Quality of life improvement is among the benefits from this agent in patients with congenital disease.
A retrospective observational study's clearest takeaway was that the agent can reduce spontaneous bleeding in severe disease.
These proof-of-concept data may spur further studies toward developing a single-intervention cure for patients.
Patients with hemophilia A who received prophylaxis had a reduced risk for ICH.
Analysis shows pharmacokinetic-guided dosing may be effective for guiding prophylactic factor concentrate therapy.
Compared with before surgery, the percentage of factor VIII increase was 42.9% at day one and 36.4% on day two.
Free tissue factor pathway inhibitor levels were associated with thrombin generation in patients with severe hemophilia.
The US FDA has approved fidanacogene elaparvovec-dzkt, a gene therapy for adults with hemophilia B.
Dr. Mamauag details her study on the need for factor VIII infusions in patients with tolerized hemophilia A on emicizumab.
The US FDA has approved an expanded indication for IXINITY to control bleeding in all patients with hemophilia B.
Desmopressin with FVIII during operations was reportedly feasible and safe in patients with nonsevere hemophilia A.
Patients with hemophilia A or B had low rates of VTE after total joint replacement despite infrequent thromboprophylaxis use.
Desmopressin was safe and effective for women with inherited bleeding disorders during both pregnancy and delivery.
Health Canada approved fidanacogene elaparvovec, an adenoviral vector-based gene therapy for patients with hemophilia B.
The FDA and EMA are expected to make decisions on once-weekly subcutaneous marstacimab for patients with hemophilia A or B.
Large deletions in the FVIII gene (F8) were shown to be predictors of ITI therapy failure.
Emicizumab plus ITI did not induce new safety signals in patients with hemophilia A.
A single infusion of AAV-HLP-hFVIII-V3 induced stable FVIII expression over five years.
Women with inherited bleeding disorders may need prophylaxis.
First patient treated with hemophilia A gene therapy demonstrates "promising" outcomes.
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