FDA Partially Rescinds Indications for BeneFIX and Ixinity
Owing to conflicting market exclusivity rights, the U.S. Food and Drug Administration (FDA) has partially rescinded its decision to approve…
Articles by Joana Carvalho, PhD
Efanesoctocog Alfa on Fast Track for Hemophilia A
The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII…
Preventive Advate Found Better Than On-demand Treatment in Hemophilia A
When given as a prophylactic, or preventive therapy, Advate worked better than on-demand treatment at lowering the number of…
Gene Therapy SPK-8016 Shows Early Promise for Severe Hemophilia A
A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of…
Hemlibra Lowered Number of Hemophilia A Bleeds Over Nearly 6 Years
When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of…
Top 10 Hemophilia Stories of 2020
Hemophilia News Today brought you daily coverage in 2020 of important discoveries, treatment advancements, clinical trial findings, and other important…
FLT180a Gene Therapy Can Prevent Bleeds in Hemophilia B Patients for Up to 3 Years, Data Show
A single dose of the experimental gene therapy FLT180a can safely maintain the activity levels of factor IX (FIX)…
Interviews Reveal Women With Genetic Blood Disorders Share Uncertainties Regarding Diagnosis
Women with genetic blood disorders, including those who are carriers of hemophilia A and B, face multiple uncertainties…
CRISPR/Cas9 May Be Better Than Gene Therapy at Restoring FIX Activity
Note: This story was updated Nov. 6, 2020, to clarify key scientific concepts. The gene-editing tool CRISPR/Cas9 enables…
Roche Proposes Transfer Needle With Filter When Using Hemlibra
Reports of translucent particles of being detected in Hemlibra (emicizumab) in 2019 do not pose a safety risk…