Results 101 to 110 of about 46,727 (267)
ELEVATED MEAN CELL VOLUME IN SICKLE CELL ANAEMIA: ONE STORY, TOO MANY? [PDF]
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
Neurodevelopmental screening in toddlers or preschoolers with sickle cell disease (SCD) predicts future academic deficits. Screening sensitivity for future academic deficits is highest in preschoolers with SCD. Brief neurodevelopmental screening tools can identify high risk children for early intervention. Abstract Aim To assess the predictive validity
Sarah E. Bills +5 more
wiley +1 more source
ABSTRACT In the phase 2, double‐blind, randomized controlled BEYOND trial (NCT03342404), luspatercept increased hemoglobin levels in patients with non‐transfusion‐dependent β‐thalassemia (NTDT). This study assessed long‐term effects of luspatercept on patient‐reported outcomes (PROs), using data from BEYOND and patients who continued luspatercept ...
Khaled M. Musallam +12 more
wiley +1 more source
The inheritance of the sickle gene (hemoglobin S) and another abnormal hemoglobin gene is described as sickle cell disease, and the homozygous form of the disease is hemoglobin SS.
John A Ashindoitiang +4 more
doaj +1 more source
Background Human induced pluripotent stem cells offer perspectives for cell therapy and research models for diseases. We applied this approach to the normal and pathological erythroid differentiation model by establishing induced pluripotent stem cells ...
Ladan Kobari +15 more
doaj +1 more source
Inherited hemolytic disorders with high occurrence of b-thalassemia in Sindhi community of Jabalpur town in Madhya Pradesh, India [PDF]
Hereditary hemolytic disorders such as hemoglobin disorders, β-thalassemia syndrome, G6PD deficiency, and ABO and Rhesus blood groups are the most common public health problems in India.
Balgir, RS
core +1 more source
Gene Editing for Haemophilia—The Next Frontier
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
Hair‐on‐end sign in severe sickle cell disease
British Journal of Haematology, EarlyView.
Raquel da Costa Neves +2 more
wiley +1 more source

