Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A
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
Effects of exercise interventions on health-related quality of life in older adults with osteoporosis: a systematic review and meta-analysis. [PDF]
Kenzhegazova G +7 more
europepmc +1 more source
Abstract Objectives We examined whether discrepancies between self‐reported and parent proxy–reported health‐related quality of life (HRQoL) were associated with child well‐being in children and adolescents attending a headache clinic compared to controls.
Amalie Akulenok Berring‐Uldum +2 more
wiley +1 more source
Health-related quality of life and physical activity in children with Multiple Osteochondromas. [PDF]
Amajjar I +4 more
europepmc +1 more source
Background Credible long‐term outcomes from randomized trials evaluating the effectiveness and cost‐effectiveness of preventive programs for mental health problems are needed. We compared long‐term effects of the Mind My Mind (MMM) transdiagnostic cognitive behavioral therapy (CBT) program to management as usual (MAU). Methods The study was a pragmatic,
Ditte Vassard +14 more
wiley +1 more source
Linking physical activity to health-related quality of life among middle-aged adults: the mediating roles of resilience and physical exercise self-efficacy. [PDF]
Yang Q, Liu B, Qin S, Huang J, Zhang Q.
europepmc +1 more source
Family quality‐of‐life burden in chronic spontaneous urticaria: A multicentre study
Poor control of chronic spontaneous urticaria (CSU) significantly worsens family members' quality of life, especially in emotional, physical, and social domains. Higher disease severity was linked to increased caregiving burden and household expenditures, highlighting the need for family‐centred CSU care and support across diverse global populations ...
Beatrice Martinez Zugaib Abdalla +33 more
wiley +1 more source
Bimekizumab efficacy and safety in Chinese patients with psoriasis in the BE SHINING Phase 3 study
In Chinese patients with moderate to severe plaque psoriasis, bimekizumab treatment was well tolerated and led to significantly higher clinical response rates versus placebo, including complete skin clearance. These findings were consistent with global studies and suggest that bimekizumab is an effective treatment option for Chinese people living with ...
Lin Cai +11 more
wiley +1 more source
From cognition to daily functioning: Insights from iatrogenic cerebral amyloid angiopathy. [PDF]
Marinoni G +12 more
europepmc +1 more source
Effects of a falls prevention exercise programme on health-related quality of life in older home care recipients: a randomised controlled trial [PDF]
Bergland, Astrid +4 more
core +1 more source

