Results 61 to 70 of about 31,702 (234)
Bringing Gene Therapy Into Real World Clinical Practice
Haemophilia, EarlyView.ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach +2 more
wiley +1 more source
Clinical Epidemiology and Global HealthObjective: The treatment of Hemophilia A and B has significantly evolved. Study design: we conducted a retrospective, observational study among patients with HA and HB managed in a Pediatric Hematology and Oncology Unit for almost 22 years for a total of
M.P. Esposto +10 more
doaj +1 more source
FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives
Haemophilia, EarlyView.ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita +2 more
wiley +1 more source
Gene Editing for Haemophilia—The Next Frontier
Haemophilia, EarlyView.ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti +3 more
wiley +1 more source
Performing Large‐Scale Genetic Analysis in the Bleeding Disorders Community
Haemophilia, EarlyView.ABSTRACT Inherited bleeding disorders encompass a diverse group of conditions caused by genetic defects affecting coagulation factors, fibrinogen, von Willebrand factor, or platelet function. Despite major advances in quantitative and functional laboratory assays, a substantial diagnostic gap remains, particularly in patients with mild or atypical ...
Anna R. Blankstein +6 more
wiley +1 more source
Haemophilia, EarlyView.ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini +4 more
wiley +1 more source
Haemophilia, EarlyView.ABSTRACT Introduction Women with haemophilia can present manifestations and experience undiagnosed symptoms, which negatively affect their health‐related quality of life (HRQoL). Objective To evaluate the HRQoL in obligate carriers of haemophilia (OCH) versus women non‐carriers of haemophilia (WNCH) in a Colombian population.
Fabian A. Manzano‐Di Zeo +9 more
wiley +1 more source
The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B
Blood Advances, 2018 : Data are needed on minimal factor activity (FA) levels required to prevent bleeding in hemophilia. We aimed to evaluate associations between hemophilia type and FA level and joint bleeding and orthopedic procedures using longitudinal data.
J. Michael Soucie +4 more
doaj +1 more source
Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A
Haemophilia, EarlyView.ABSTRACT Introduction Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5 (AAV5)‐mediated gene therapy for severe haemophilia A (HA). Aim Report the final safety and efficacy results of the phase 1/2 trial of valoctocogene roxaparvovec. Methods An open‐label phase 1/2 trial (NCT02576795) enrolled adult males with severe HA (factor
Priyanka Raheja +9 more
wiley +1 more source