Results 91 to 100 of about 204,109 (254)

Identification of predictive factors for reversal of cerebral vasculopathy in an original longitudinal cohort study in newborns with sickle cell anaemia

British Journal of Haematology, EarlyView.
Summary Cerebral macrovasculopathy (CV) is a major complication in children with sickle cell anaemia (SCA) and usually requires a long‐term transfusion programme (TP) to prevent stroke. This study aimed to identify factors predicting reversal of CV on TP in a single‐centre newborn cohort. Among 375 patients, 50 presented CV and received TP.
Julie Sommet, Priscilla Boizeau, Zinedine Haouari, Berengère Koehl, Monique Elmaleh, Emmanuelle Lesprit, Florence Missud, Ghislaine Ithier, Laurent Holvoet, Valentine Brousse, Christophe Delclaux, André Denjean, Suzanne Verlhac, Corinne Alberti, André Baruchel, Nathalie Couque, Malika Benkerrou   +16 more
wiley   +1 more source

Covalent drug discovery: Progress against key targets, emerging strategies and lessons learnt

British Journal of Pharmacology, EarlyView.
Abstract Covalent drug discovery is currently experiencing a boom in industrial and academic interest. To date, at least 75 covalent drugs have received regulatory approval, targeting both traditional target classes and more challenging proteins for which other approaches failed. In many cases, unique aspects of covalent targeting are essential for the
Charles P. Brown, Alan Armstrong, David J. Mann   +2 more
wiley   +1 more source

Targeting protein–protein interactions with reversible covalent modalities: Non‐cysteine chemistries

British Journal of Pharmacology, EarlyView.
Abstract Protein–protein interactions (PPIs) are central to diverse cellular functions, and represent a rapidly expanding class of therapeutic targets. Advancements in covalent drug design have enabled small‐molecule drugs to overcome challenges associated with engaging these targets, such as limited durations of action and difficult‐to‐drug (expansive,
Ruchira Basu, Steven Fletcher
wiley   +1 more source

Safety of Lowering the Platelet Transfusion Threshold Before Central Venous Catheterisation in Patients With Haematological Diseases

European Journal of Haematology, EarlyView.
ABSTRACT Introduction Central venous catheter (CVC) insertion is frequently required in patients with haematological diseases. Despite limited evidence, thrombocytopenia often prompts prophylactic platelet transfusion before catheterisation. Methods We conducted an observational before‐and‐after study including the first non‐tunnelled CVC insertion ...
Mathias Lazarevic Lindblad, Elin Pålsson, Vladimir Lazarevic, Leila Naddi, Ola Borgquist, Lars Nilsson, Maria Adrian, Björn P. Bark, Thomas Kander   +8 more
wiley   +1 more source

Molecular dynamics simulations elucidate the misfolding mechanisms of secretion‐defective pancreatic lipase variants

The FEBS Journal, EarlyView.
Misfolding mutations in pancreatic lipase have been identified as potential contributors of chronic pancreatitis, an inflammatory disease of the human pancreas. Here, we describe the effect of these misfolding mutations on pancreatic lipase structure using molecular dynamics simulations and structural modeling.
Gyula Hoffka, András Szabó
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

Determining of the Hydroxyurea Effluence on TCD Parameters of Sickle Cell Patients

مجله دانشکده پزشکی اصفهان, 2009
Background: Sickle cell disease is the most common hemoglobinopathies that affect a specific intracellular protein named as hemoglobin. This disease has several clinical manifestations including, CNS involvement.
Nasser Sharafadinzadeh, Nastaran Majdinasb, Khodamorad Zandian, Keiwan Taherpoor, Armaghan Moravej Aleali, Mohamad Pipelzadeh   +5 more
doaj  

ELEVATED MEAN CELL VOLUME IN SICKLE CELL ANAEMIA: ONE STORY, TOO MANY? [PDF]

Sanamed
Introduction: Sickle cell disease is a hereditary blood disorder characterized by defective hemoglobin. Red cell indices are proposed as potential tools for diagnosing and managing sickle cell disorders.
Jeremiah Zaccheaus, Alee Magnus
doaj   +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

Ocular Complications in Adults with Sickle Cell Disease in Lagos – A Comparative Study

Nigerian Journal of Medicine
Introduction: Sickle cell disease has been associated with ocular complications in several studies. However, it has not often been compared with nonhemoglobinopathy controls.
Kuburat Oliyide, Olubanke Theodora Ilo, Olufemi Emmanuel Babalola, Tunji Adenekan, Michael Olufemi Kehinde, Aribaba Olufisayo Tade, Folasade Akinsola   +6 more
doaj   +1 more source