Starting immune tolerance induction early linked to treatment success
For children with severe hemophilia A who develop inhibitors — neutralizing antibodies that reduce the effectiveness of standard therapies…
Articles by Marisa Wexler, MS
Decision on hemophilia A gene therapy Roctavian set for summer
The U.S. Food and Drug Administration (FDA) is extending its review of BioMarin Pharmaceutical’s request to approve the gene…
Most hemophilia A children were bleed-free after a year on Altuviiio
Most children with hemophilia A given Altuviiio (efanesoctocog alfa) once a week were bleed-free throughout about…
Efanesoctocog Alfa Reduces Bleeding, Pain in Phase 3 Study
Most people with severe hemophilia A who were given once-weekly treatment with efanesoctocog alfa in the Phase 3…
WFH Launches Global Registry to Monitor Gene Therapy Outcomes
The World Federation of Hemophilia (WFH) has launched a registry to monitor long-term outcomes of safety and effectiveness for people…
Gene Therapy Reduces Bleeding in Men With Hemophilia B
Treatment with the experimental gene therapy fidanacogene elaparvovec led to significant reductions in bleeding rates for men with moderately severe…
Most Patients Bleed-free at 3 Years After Roctavian in GENEr8-1 Trial
Three years after receiving the one-time gene therapy Roctavian (valoctocogene roxaparvovec) at trial, the majority of men with severe…
Long-term Data Reinforce SPK-8011 Gene Therapy’s Potential for Hem A
One-time treatment with the investigational gene therapy SPK-8011 led to sustained low bleed rates for people with hemophilia A…
HEM B Therapy Etranacogene Dezaparvovec Wins Thumbs-up
A committee of the European Medicines Agency (EMA) has issued a positive opinion recommending the one-time gene therapy etranacogene…
Inspection of Roctavian Facility Set; FDA Seeks More Trial Data
The U.S. Food and Drug Administration (FDA) has scheduled its inspection of the facility in California where BioMarin Pharmaceutical…