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.”

An application to the U.S. Food and Drug Administration is expected to be supported by data that showed risto-cel's benefit in 31 patients. As a medical scientist for 18 years, he worked in both academia and industry, where his research focused on the discovery of new vaccines and medicines to treat inflammatory disorders and infectious diseases. Steve is a published author in multiple peer-reviewed scientific journals and a patented inventor. Gene-edited cell therapy U.S. Beam Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to approve ristoglogene autogetemcel, or risto-cel, its single-dose, gene-edited cell therapy candidate for people with sickle cell disease (SCD), as early as year’s end. Disease burden heavy for SCD patients in US, despite promise of gene therapy Base editing is a precise gene-editing technology that can create a change in the DNA’s sequence without cutting through both side rails of the DNA’s twisted ladder, a characteristic structure known as its double helix. Such cuts can damage DNA and create unwanted genetic abnormalities. Interim trial data is described in the study “Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease,” which was published in The New England Journal of Medicine.

Original title: “Beam to seek OK of gene-edited cell therapy risto-cel for sickle cell disease”