Read twice and referred to the Committee on Health, Education, Labor, and Pensions.

Original title: “S 735: Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2025”

Referred to the House Committee on Energy and Commerce.

Original title: “HR 1796: Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2025”

Original title: “Charter Amendments, Establishments, Reestablishments, Renewals, and Terminations; Sickle Cell Advisory Committee”

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients. This decision follows a full submission assessed through the SMC orphan medicine process and applies only in the context of an approved NHS Scotland Patient Access Scheme, which allows confidential pricing arrangements for high-cost therapies. SCD is a chronic, inherited blood disorder caused by mutations in the beta-globin gene, leading to the production of sickle haemoglobin. Clinical experts highlighted its potential to deliver durable disease modification after a single intervention, reducing dependence on hydroxycarbamide, blood transfusions, and recurrent hospital care. Exagamglogene autotemcel is a personalised, autologous, cell-based gene therapy that uses CRISPR/Cas9 gene-editing technology to modify a patient’s own stem cells. All patients experienced treatment-related adverse events, which were primarily attributable to busulfan conditioning treatment rather than to the gene therapy itself. Common events included febrile neutropenia, infections, and cytopenias, typically occurring within the first 6 months after infusion. No cases of graft failure or graft-vs-host disease were reported. Cite this: Edited by Zeel Mehta. Scotland Approves Gene Therapy for Sickle Cell Disease - Medscape - January 20, 2026.

Original title: “Scotland Approves Gene Therapy for Sickle Cell Disease”

compound heterozygosity for hemoglobin s and d punjab an interplay of two haemoglobinopathies rare mimic of sickle cell disease

Original title: “Compound Heterozygosity for Hemoglobin S and D-Punjab: An Interplay of Two Haemoglobinopathies/Rare Mimic of Sickle Cell Disease.”

New supplement from the American Journal of Preventive Medicine describes the state of sickle cell disease related care in the United States.

Original title: “Advancements in Sickle Cell Disease - Media Advisory”

The Advisory Committee for Immunization Practices voted today 13 to 1, with 1 abstention, to recommend that infants at increased risk for meningococcal disease should be vaccinated with 4 doses of HibMenCY at 2, 4, 6, and 12 through 15 months. These include infants with recognized persistent complement pathway deficiencies and infants who have anatomic or functional asplenia including sickle cell disease. HibMenCY can be used in infants ages 2 through 18 months who are in communities with serog

Original title: “CDC Advisory Committee on Immunization Practices Recommends HibMenCY for Infants at Increased Risk for Meningococcal Disease”

The first national estimate on the health status of children with sickle cell disease revealed that black children with sickle cell disease are more likely to have intellectual disabilities, hearing deficits, and frequent severe headaches or migraines than black children without sickle cell disease.

Original title: “CDC Releases Study on Children With Sickle Cell Disease”