Results 201 to 210 of about 51,427 (307)

Google searches show persistent use of outdated terminology for angioedema in Germany

open access: yes
JDDG: Journal der Deutschen Dermatologischen Gesellschaft, EarlyView.
Sophia Neisinger   +3 more
wiley   +1 more source

Safety and Efficacy of Damoctocog Alfa Pegol Prophylaxis in Patients With Severe Haemophilia A: Results of an Interventional, Post‐Marketing Study

open access: yesHaemophilia, EarlyView.
ABSTRACT This is a plain language summary of the results of a trial of damoctocog alfa pegol (BAY 94–9027, Jivi).
Pål André Holme   +5 more
wiley   +1 more source

Semaglutide and Cardiovascular Outcomes According to Baseline Aspirin Use: Analyses of the SOUL and SELECT Trials

open access: yes
Diabetes, Obesity and Metabolism, EarlyView.
Dirk Müller‐Wieland   +11 more
wiley   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

open access: yesHaemophilia, 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

FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives

open access: yesHaemophilia, 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

open access: yesHaemophilia, 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

Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A

open access: yesHaemophilia, 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

The Swiss Haemophilia Registry–Report From the First 8 Years

open access: yesHaemophilia, EarlyView.
ABSTRACT Introduction Patient registries capture disease related information and provide a valuable source for real‐world data on rare diseases and their management. The Swiss Haemophilia Registry (SHR) was established in 2015 on the basis of a new Swiss federal human research act.
Alessandra Bosch   +8 more
wiley   +1 more source

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