Hemophilia
BE-101 is a single IV infusion which does not require preconditioning or immunosuppression in recipients.
Concizumab-mtci improves thrombin production by blocking the tissue factor pathway inhibitor protein.
In the phase 3 explorer8 trial, concizumab reduced the bleeding rate in patients with hemophilia A and B.
Annette Von Drygalski, MD, PharmD, discussed the promising role of etranacogene dezaparvovec gene therapy in hemophilia B.
In a cohort of adults and adolescents marstacimab produced bleed control superior to that from on-demand bypassing agent.
Dr. John Gansner and Dr. Martina H. Slingsby argue this new agent can provide benefit across all bleeding disorders.
Heme Today spoke with Ulrike Reiss, MD, and Andrew Davidoff, MD, of the trial's investigator team.
A preclinical mouse study showed hemostasis restoration, as well as use for active bleeding and internal bleeding prevention.
Fidanacogene elaparvovec gene therapy is a safe and effective treatment for patients with hemophilia B, research shows.
Approval was based on data from phase 3 studies in the ongoing ATLAS clinical development program.
The Clinical Practice Guidance document issued by the EHA, ISTH, EAHAD, and ESO addresses both hemophilia A and B.
A study cohort with severe disease over a median follow-up of 14 months had an annualized bleeding rate of zero.
This approval for patients aged 12 years and older was based on findings from the explorer7 phase III clinical trial.
A new study addresses the need for data on treatment outcomes in patients younger than 12 years of age.
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.
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