Results 111 to 120 of about 3,402 (214)

Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies

British Journal of Haematology, EarlyView.
Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi, Nandakumar Natarajan, Kathryn E. Dickerson, Richard A. Voit   +3 more
wiley   +1 more source

Predict less, prevent more: A familiar mantra for sickle cell anaemia?

British Journal of Haematology, EarlyView.
Commentary on: Sommet J, Boizeau P, Haouari Z, et al. Identification of predictive factors for reversal of cerebral vasculopathy in an original longitudinal cohort study in newborns with sickle cell anaemia. Br J Haematol. 2026 (Online ahead of print). doi: 10.1111/bjh.70726.
Charles T. Quinn
wiley   +1 more source

INtraVESical immunoTherapy (INVEST) prior to radical cystectomy for bladder cancer: trial protocol

BJU International, EarlyView.
Background High‐risk non‐muscle‐invasive bladder cancer (HRNMIBC) has a variable prognosis, managed predominantly by local surgical resection and intravesical Bacillus Calmette–Guérin (BCG), or radical cystectomy (RC). Current treatments are poorly tolerated, have supply limitations and often fail to control the disease.
Ruby Lister‐Whelan, Ethan Senior, Jessica Kendall, Jamie B. Oughton, Fiona Collinson, Jon Griffin, Rachel Hubbard, Steven Kennish, Aidan Noon, Derek J. Rosario, Irbaz B. Riaz, Julie Telliez, James W. F. Catto, Syed A. Hussain   +13 more
wiley   +1 more source

The role of protein arginine methyltransferases in sickle cell‐mediated neurovascular impairments in mice

British Journal of Pharmacology, EarlyView.
Background and Purpose Sickle cell disease (SCD) is a hereditary blood disorder caused by mutation of β‐globin. In SCD, haemoglobin polymerization causes red blood cells to assume a rigid sickle‐shape leading to neurovascular alterations, including development of vascular dementia – characterized by cognitive impairment and reduced cerebral perfusion ...
Julia Zaccarelli‐Magalhães, Mariana Sayuri Berto Udo, Drew James Smith, Julia Langman, Luiz Henrique Matuguma, Guilherme Nagamine Gomes, Peter Rodriguez, Ammar Husan, Quanguang Zhang, Christopher G. Kevil, Cristiane Teresinha Citadin, Hung Wen Lin   +11 more
wiley   +1 more source

Paediatric‐onset autoimmune cytopenia: How can we reduce the long‐term mortality?

British Journal of Haematology, EarlyView.
Nathalie Aladjidi, Thierry Leblanc, Helder Fernandes, Capucine Picard, Jacinta Bustamante, Frédéric Rieux‐Laucat, Mathieu Fusaro, Maud Tusseau, Charlotte Durand‐Teyssier, Emma Cohendy, Jérôme Granel, Stéphane Ducassou, Agathe Escudier, Despina Moshous, Marc Michel, Bertrand Godeau, Sébastien Héritier, Mony Fahd, Brigitte Bader‐Meunier, Alain Fischer, Guy Leverger, All collaborators from the French Paediatric Haemato‐Immunologic CEREVANCE Network   +21 more
wiley   +1 more source

Inborn errors of immunity in children with neuroinflammation

Developmental Medicine &Child Neurology, EarlyView.
Abstract Inborn errors of immunity (IEIs), an expanding group of monogenic disorders with diverse clinical manifestations, are increasingly recognized to include neuroinflammatory disease. Examples of diseases included under this umbrella are Aicardi–Goutières syndrome, deficiency of adenosine deaminase 2, familial haemophagocytic lymphohistiocytosis ...
Eppie M Yiu, Dilan Dissanayake, Jessie Cunningham, Helen Branson, Ori Scott, E Ann Yeh   +5 more
wiley   +1 more source

Dose–Response Association Between Systemic and Dermatologic Glucocorticoid Use and Type 2 Diabetes Mellitus: A Nationwide Case–Control Study

Diabetes, Obesity and Metabolism, EarlyView.
ABSTRACT Aims To investigate the association and dose–response between systemic and topical glucocorticoids and odds of Type 2 diabetes mellitus. Materials and Methods We conducted a nationwide case–control study using Danish registry data from 2013 to 2021. People aged 40 years or older with incident type 2 diabetes mellitus (n = 149 113) were matched
David Vadsholt, Annika V. Kvist, Chano Sandiramoorthy, Joshua R. Lewis, Peter Vestergaard, Johan Røikjer, Nicklas H. Rasmussen   +6 more
wiley   +1 more source

Pegcetacoplan Delivers Real‐World Therapeutic Benefits and Reduces Disease Burden for Patients With Paroxysmal Nocturnal Haemoglobinuria: A Systematic Literature Review of Pegcetacoplan Real‐World Clinical and Patient‐Reported Outcomes

European Journal of Haematology, EarlyView.
ABSTRACT Aims Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra‐rare, acquired, non‐malignant haematological disorder that, if left untreated, can lead to significant morbidity. This systematic literature review (SLR) summarized real‐world evidence (RWE) for pegcetacoplan, a complement 3/3b inhibitor (C3i) available since 2021.
Juan Carlos Vallejo Llamas, Koo Wilson, Zalmai Hakimi, Vikte Lionikaite, Elisabetta Metafuni   +4 more
wiley   +1 more source

Is There a Difference in Occurrence of Complications Between Adults With Hemoglobin SS and Hemoglobin SC Disease: An Extended Systematic Review

European Journal of Haematology, EarlyView.
ABSTRACT Sickle cell disease (SCD) is characterized by both acute and chronic complications. The clinical manifestation of these complications differs between genotypes. Given the large amount of research already published, this systematic review aims to offer a complete overview of types of sickle cell complications between adults in the most common ...
Martijn van der Meer, Kim Velsink, Wing H. Tong, Jan W. Schoones   +3 more
wiley   +1 more source

Clinical predictors of mortality in immune thrombotic thrombocytopenic purpura: A National Inpatient Sample analysis

British Journal of Haematology, EarlyView.
Anand Shah, Christopher Chen, Ilana Pyatetsky, Shruti Chaturvedi, Ankit Shah   +4 more
wiley   +1 more source