Results 171 to 180 of about 47,631 (258)

Randomised Controlled Feasibility Trial of Face‐To‐Face Diabetes Self‐Management Education Shows High Completion Rates Are Needed to Improve Patient‐Reported Outcomes

Diabetes, Obesity and Metabolism, EarlyView.
ABSTRACT Aims To examine the dose–response relationship between diabetes self‐management education (DSME) attendance and psychological outcomes in type 2 diabetes, assessing whether minimal attendance (10%) produces clinically meaningful improvements and comparing outcomes at the internationally adopted 60% completion benchmark with full (100 ...
Gemma A. Lewis, David M. Hughes, Greg J. Irving, John P. Wilding, Kevin J. Hardy   +4 more
wiley   +1 more source

How the First 9 Months Shape the Rest of Your Life: The Impact of Gestational Diabetes on the Metabolic Future

Diabetes, Obesity and Metabolism, EarlyView.
ABSTRACT Gestational diabetes mellitus (GDM) represents far more than a transient glycaemic disorder: it is a sentinel metabolic event that unveils pre‐existing defects in β‐cell function and insulin action, with profound implications for both maternal and offspring cardiometabolic health across the lifespan.
Mustafa Kanat, Muhammad Abdul‐Ghani, Ralph A. DeFronzo   +2 more
wiley   +1 more source

Project Sickle Cure: A Prospective, International Observational Study of Hematopoietic Cell Transplantation for Sickle Cell Disease

European Journal of Haematology, EarlyView.
ABSTRACT Background Sickle cell disease (SCD) is a chronic and life‐limiting hemoglobin and systemic vascular disease. While over 1000 people have undergone hematopoietic cell transplantation (HCT) over the last 40 years, long‐term disease‐specific and health‐related quality of life data are lacking.
Gregory M. T. Guilcher, Allistair Abraham, Staci D. Arnold, Christine Camacho‐Bydume, Jenny Duong, Scott Gillespie, Ann Haight, Monica L. Hulbert, Naresh Kumar, Katie Liu, Cherry Mammen, Aleksandra Mineyko, Abra Morgan, Alexander Ngwube, Robert S. Nickel, Araby Roberts, Fiona Schulte, Stephen Sands, Akshay Sharma, John T. Horan, Elizabeth O. Stenger   +20 more
wiley   +1 more source

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, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

Lessons from clinical and genetic characterization of intellectual disability

Developmental Medicine &Child Neurology, EarlyView.
Fuki Marie Hisama
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, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +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, Sterre P. E. Willems, Saskia E. M. Schols, Rosanna Asselta, Gillian Lowe, Neil V. Morgan, Paula D. James   +6 more
wiley   +1 more source

Seventh Åland Island Meeting on von Willebrand Disease

Haemophilia, EarlyView.
ABSTRACT Introduction The seventh Åland Island Meeting on von Willebrand Disease (VWD) was held on the Åland archipelago in Finland, from 26 to 28 September 2024. Aim The meeting brought together experts in the field of VWD from around the world to share the latest advances and knowledge in VWD.
Riitta Lassila, Ruben Bierings, Nathan T. Connell, Fernando F. Corrales‐Medina, Augusto B. Federici, Veronica H. Flood, Floor Heubel‐Moenen, Jill M. Johnsen, Geoffrey Kuppens, Davide Matino, Carolyn Millar, Ingmar Schoen, Anish Sharda, Robert F. Sidonio Jr, Alok Srivastava, Timea Szanto, Angela C. Weyand, Erik Berntorp   +17 more
wiley   +1 more source

Patient-derived induced pluripotent stem cells for precision modelling of monogenic beta cell disorders. [PDF]

Front Endocrinol (Lausanne)
Deng L, Krishnamurthy M.
europepmc   +1 more source

Australian clinical practice guideline: diagnosis and treatment of idiopathic multicentric Castleman disease

Internal Medicine Journal, EarlyView.
Abstract Idiopathic multicentric Castleman disease (iMCD) is a rare condition. The pathogenesis is incompletely understood; however, interleukin‐6 (IL‐6) is a major mediator. The clinical presentation is heterogeneous, from mild constitutional symptoms to severe multi‐organ failure.
Dipti Talaulikar, Jun Yen Ng, Adam Bryant, Annmarie Bosco, Hugh C. Caterson, Pratyush Giri, Pravin Hissaria, Simon J. Harrison, Stephen Lade, Connull Leslie, Jacinta Perram, H. Miles Prince, Dejan Radeski, Maureen Rischmueller, Craig Wallington‐Gates, Nicholas Weber, Alberta Hoi   +16 more
wiley   +1 more source