Brief electrical pulse may help in hemophilia gene therapies: Study
Gentle electrical stimulation may help liver cells uptake gene therapies, such as those used to treat hemophilia, according to…
Articles by Lindsey Shapiro, PhD
FDA OK’s virus antibody test to determine Beqvez eligibility
The U.S. Food and Drug Administration (FDA) has approved Labcorp’s companion diagnostic test for detecting preexisting antibodies against the viral…
FDA approves Beqvez gene therapy for adults with hemophilia B
The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy fidanacogene elaparvovec for treating certain adults…
Ixinity now OK’d for hemophilia B children younger than 12 in US
Ixinity (trenonacog alfa) has been approved by the U.S. Food and Drug Administration (FDA) for use in children younger…
Intellia ending work with Regeneron on gene therapy for hemophilia B
Note: This story was updated March 28, 2024, to clarify the gene therapy delivers the mRNA of the Cas9 enzyme.
SerpinPC eases joint involvement in severe hemophilia: Trial data
Almost three years of treatment with SerpinPC was associated with a nearly complete resolution of bleeds and a…
Blocking anticoagulant protein S may boost replacement therapy
Adding an antibody targeting the anticoagulant protein S to factor IX (FIX) replacement therapy accelerated blood clotting in samples…
Health Canada approves Hemgenix gene therapy for hemophilia B
Health Canada has approved the gene therapy Hemgenix (etranacogene dezaparvovec) for treating adults with hemophilia B who rely…
Joint impairments linked to functional decline in mild hemophilia
Impairments in joint health are associated with certain functional deficits in the lower limbs among people with mild hemophilia,…
Long-term Eloctate found to reduce all bleed types in Phase 3 analyses
Preventive treatment with Eloctate (efmoroctocog alfa) for up to five years led to sustained reductions in all bleed types…