Sickle Cell Disease news and research updates
Research readouts, treatment and access decisions, and community updates for people living with sickle cell disease and the people who care for them — each one dated, with a link to the original source where there is one.
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Agios Pharmaceuticals to Present RISE UP Phase 3 Trial Results of Mitapivat in Sickle Cell Disease at EHA 2026 Plenary Session
Agios Pharmaceuticals will present Phase 3 trial results for mitapivat in sickle cell disease at EHA 2026. Quiver AI Summary Agios Pharmaceuticals announced that detailed results…
Newer blood thinners tied to less bleeding in sickle cell disease study
Newer oral anticoagulants, or blood thinners, were linked to a lower risk of clinically relevant nonmajor bleeding than warfarin, an older blood thinner, in adults with sickle…
Beam Therapeutics to Present Updated Biomarker Data from Phase 1/2 BEACON Trial Further Underscoring Risto-cel’s Ability to Restore Red Blood Cell Health and Function in Sickle Cell Disease at EHA2026
CAMBRIDGE, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing…
Red Blood Cell Exchange in Sickle Cell Disease Care: A Comprehensive Review
ABSTRACT Erythrocytaphoresis or RBC exchange (RBCX) is a key intervention in the management of sickle cell anemia for both chronic prophylaxis and emergent clinical care during…
Hematopoietic Stem Cell Transplantation as Curative Therapy in Sickle Cell Disease
Sickle cell disease (SCD) is a hereditary hemoglobinopathy characterized by chronic hemolytic anemia, recurrent vaso-occlusive crises, progressive organ damage, and reduced life…
Agios Submits sNDA to FDA for U.S. Accelerated Approval of Mitapivat in Sickle Cell Disease
CAMBRIDGE, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative…
Implementation of third-generation digital PCR for non-invasive prenatal diagnosis of sickle cell disease and early detection. Pilot study
Sickle cell disease (SCD) is one of the most prevalent autosomal recessive conditions worldwide, affecting more than 600,000 newborns annually. Despite advances in treatment, it…
HCT Shows Promising Safety and Efficacy in SCD
HLA-matched sibling donor (MSD) hematopoietic cell transplantation (HCT) done prior to adolescence demonstrated promising safety and efficacy outcomes among patients with sickle…
Frailty phenotype in adults with sickle cell disease
Abstract Background: People with sickle cell disease (SCD) are at risk for accelerated biological aging and functional decline due to both age and SCD-related stressors. Frailty…
Sustainment of Hydroxyurea Adherence in Patients With Sickle Cell Disease
Key Points Question Do hydroxyurea use (ie, taking the medication at all) and adherence (ie, days taken per week) change over time, and are they associated with patient…
Genetic, clinical, and biological risk factors associated with optic nerve thinning in sickle cell disease
Objectives To determine whether adults with sickle cell disease (SCD) without glaucoma exhibit subclinical structural optic neuropathy (SON) on optical coherence tomography (OCT)…
Understanding Sickle Cell Disease Endothelial Pathology Through the Lens of Liver Sinusoidal Endothelial Cells
What are the main findings? Endothelial dysfunction is a hallmark of sickle cell disease (SCD), which is associated with both acute and chronic crises in patients. Whereas lung or…