Results 181 to 190 of about 52,285 (258)

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

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

Will Extended‐Valency Pneumococcal Conjugate Vaccines Offer Enhanced Coverage Against Invasive Pneumococcal Disease for At‐Risk Children?

open access: yes
Acta Paediatrica, EarlyView.
Robert Cohen   +8 more
wiley   +1 more source

Italian Patients Journey for Gene Therapy in Haemophilia A

open access: yesHaemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno   +19 more
wiley   +1 more source

Targeted liquid chromatography–tandem mass spectrometry profiling of arginine and histidine derivatives in chronic migraine: Discriminatory performance and changes after onabotulinumtoxinA therapy

open access: yesHeadache: The Journal of Head and Face Pain, EarlyView.
Plain Language Summary People with chronic migraine may have changes in amino‐acid metabolism, but it remains unclear which metabolic pathways are associated with the disease and early response to onabotulinumtoxinA treatment. We measured plasma amino‐acid levels in patients with chronic migraine and healthy controls and assessed changes after 1 month ...
Seyma Dumur   +6 more
wiley   +1 more source

Legionella pneumophila pneumonia in South Eastern Sydney: diagnosis, prognosis and public health outcomes

open access: yesInternal Medicine Journal, EarlyView.
Abstract Background Legionellosis causes more severe disease than other aetiologies of community‐acquired pneumonia (CAP). Early recognition and treatment of Legionella pneumonia reduce mortality. Urinary antigen testing (UAT) has facilitated rapid and reliable diagnosis of Legionella pneumonia. Aims The aims of this study were as follows: (i) identify
Sue Qian   +9 more
wiley   +1 more source

A spatiotemporal analysis of Salmonella bacteraemia in Queensland, 2010–2019

open access: yesInternal Medicine Journal, EarlyView.
Abstract Background Salmonellosis contributes to a significant burden on global healthcare systems. The impact of this foodborne bacterium is more pronounced when the infection is invasive, leading to bacteraemia and sepsis. Studies on invasive salmonellosis are few, and there is an urgent need to understand the spatial distribution of bacteraemia and ...
Naveen Manchal   +3 more
wiley   +1 more source

Characterisation of HIV‐1 Gag Cytotoxic T‐Lymphocyte Epitopes in the Southern African Region—A Systematic Review

open access: yesImmunology, EarlyView.
Distribution of studies on HIV‐1 Gag cytotoxic T‐lymphocyte epitopes in the southern African region showing that the majority of studies (58.44%) were conducted in South Africa, followed by Botswana (15.58%), Zambia (10.39%), Malawi (7.79%), Zimbabwe (6.49%), and Angola (1.30%).
Tumelo L. Fortuin   +4 more
wiley   +1 more source

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