Hemophilia
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.
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