Once-weekly Efanesoctocog Alfa Prevents Bleeds in Severe Hem A
Weekly use of the experimental, long-acting therapy efanesoctocog alfa safely and effectively prevents bleeds in adolescents and adults with…
Articles by Marta Figueiredo, PhD
Vibrating Microbubbles May Improve Gene Therapy Delivery for Hem A
An ultrasound-mediated, non-viral gene therapy safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting factor…
Dexamethasone After Gene Therapy May Improve Effectiveness in Hem B
Dexamethasone, a steroid with immunosuppressive and anti-inflammatory properties, may temporarily increase the efficacy of one-time gene therapy when given at…
Early Treatment Linked to Less Joint Damage in Severe Hemophilia A
In boys and men with severe hemophilia A, joint damage accumulates with age, and can be prevented if…
Cell-based Therapy Shows Promise in Mouse Model of Hemophilia A
An experimental cell-based therapy using Sernova’s innovative medical device, Cell Pouch, safely and effectively increased the levels of…
Benefits of Roctavian, Hem A Gene Therapy, Holding for 2 or More Years
A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) continues to prevent bleeds and the need for…
Top 10 Hemophilia Stories of 2021
Hemophilia News Today brought you daily coverage throughout 2021 of the latest scientific findings, treatment developments, and clinical trials related…
Gene Therapy SB-525 Controls Bleedings in Men for at Least 2 Years
A single dose of the investigational gene therapy SB-525 (giroctocogene fitelparvovec) continues to prevent bleeds and the need for prophylaxis,…
Potential Gene Therapy SPK-8011 Fares Well in Hemophilia A Study
A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing…
Catalyst Halts MarzAA Program for Hemophilia, Despite Positive Results
In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin…