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pyruvate kinase activation in the management of thalassemia and sickle cell disease
Original title: “Pyruvate Kinase Activation in the Management of Thalassemia and Sickle Cell Disease.”
rare presentation of hypersplenism in an adult female with homozygous sickle cell disease
Original title: “A rare presentation of hypersplenism in an adult female with homozygous sickle cell disease.”
cerebral fat embolism syndrome in sickle cell disease a case highlighting the predominance of susceptibility weighted imaging findings and literature review
Original title: “Cerebral fat embolism syndrome in sickle cell disease: a case highlighting the predominance of susceptibility-weighted imaging findings and literature review.”
Fulcrum’s setback is the latest in a series of stumbles for the developers of sickle cell medicines, among them Pfizer, Novartis and Intellia Therapeutics.
Original title: “Fulcrum exploring a potential sale after FDA sidelined its sickle cell drug”
Fulcrum Therapeutics’ is down more than 50% Tuesday morning as the biotech discontinues development of its lead asset. Ipsen’s Tazverik, which belongs to the same class, was pulled from the market in March due to the risk of secondary malignancies.
Original title: “Fulcrum tanks as cancer concerns stymie sickle cell asset, trigger business reorg”
study protocol for a randomized double blind placebo controlled clinical trial of desidustat oral tablet in sickle cell disease a phase iia proof of concept evaluation
Original title: “Study protocol for a randomized, double-blind, placebo-controlled clinical trial of desidustat oral tablet in sickle cell disease: a phase IIa proof-of-concept evaluation.”