PK Modulator Shows Promise in Thalassemia — In small study, mitapivat increased hemoglobin in 80% of non-transfusion-dependent patients by Mike Bassett, Staff Writer, MedPage Today August 17, 2022 ...

Because of the paucity of red blood cell precursors capable of synthesizing hemoglobin in the blood of normal people or people with thalassemia, it took a while to define the conditions in which ...

Patients with non-transfusion-dependent beta-thalassemia should be considered for interventions targeting ineffective erythropoiesis and anemia when hemoglobin levels are <10 g/dL ...

PYRUKYND® (mitapivat) net revenues; ended second quarter with $1.3 billion dollars in cash, cash equivalents and marketable securitiesPYRUKYND sNDA for thalassemia under active review, with FDA PDUFA ...

Thalassemia results from an imbalance between two proteins in hemoglobin, called alpha and beta globin. An excess of either type of protein is toxic, causing thalassemia symptoms including poor ...

The new data, presented during European Hematology Association Hybrid 2022 Congress, showed increases in fetal hemoglobin levels after a single dose of exagamglogene autotemcel, or exa-cel (CRISPR ...

Hemoglobin-S proportions of α-thalassemia-hemoglobin-S double heterozygotes were depressed. Cases of a-thalassemia-hemoglobin-E disease reported by Tuchinda et al. 10 showed similar features.

CRISPR gene editing is coming to the clinic Hematology meeting offers glimpse of clinical trials that will use CRISPR to treat sickle cell disease and beta-thalassemia beginning in 2018 by Ryan Cross ...

89% of evaluable patients (17/19) with transfusion-dependent β-thalassemia who do not have a β 0 /β 0 genotype achieved transfusion independence with 11.9 g/dL median weighted average total ...

Thalassemia, an inherited blood disorder affecting hemoglobin, has 4,361 registered patients in India, with the Health Ministry actively monitoring cases and providing financial support to the affecte ...

A patient with beta thalassemia or sickle cell disease—genetic disorders that affect the blood protein hemoglobin in red blood cells—currently has only two options for treatment. They can ...