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Genetic Modifiers of Hemoglobin Expression from a Clinical Perspective in Hemoglobinopathy Patients with Beta Thalassemia and Sickle Cell Disease.

• Authors : Diamantidis MD; Ikonomou G ...

Subjects: beta-Thalassemia*/beta-Thalassemia*/beta-Thalassemia*/genetics ; beta-Thalassemia*/beta-Thalassemia*/beta-Thalassemia*/therapy ; beta-Thalassemia*/beta-Thalassemia*/beta-Thalassemia*/metabolism

• Source: International journal of molecular sciences [Int J Mol Sci] 2024 Nov 05; Vol. 25 (22). Date of Electronic Publication: 2024 Nov 05.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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Evaluating sheep hemoglobins with MD simulations as an animal model for sickle cell disease.

• Authors : Kuczynski CE; Wake Forest Institute for Regenerative Medicine, Winston-Salem, NC, 27101, USA.; Porada CD

Subjects: Hemoglobins*/Hemoglobins*/Hemoglobins*/genetics ; Anemia, Sickle Cell*/Anemia, Sickle Cell*/Anemia, Sickle Cell*/therapy; Adult

• Source: Scientific reports [Sci Rep] 2024 Jan 02; Vol. 14 (1), pp. 276. Date of Electronic Publication: 2024 Jan 02.Publisher: Nature Publishing Group Country of Publication: England NLM ID: 101563288 Publication Model: Electronic Cited Medium: Internet ISSN:

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GBT021601 improves red blood cell health and the pathophysiology of sickle cell disease in a murine model

• Authors : Dufu, Kobina; Alt, Carsten; Strutt, Steven

Subjects: Biomedical and Clinical Sciences; Cardiovascular Medicine and Haematology; Sickle Cell Disease

• Source: British Journal of Haematology. 202(1)

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Multicenter, phase 1 study of etavopivat (FT-4202) treatment for up to 12 weeks in patients with sickle cell disease.

• Authors : Saraf, Santosh; Hagar, Robert; Idowu, Modupe

Subjects: Humans; Anemia; Sickle Cell

• Source: Blood Advances. 8(16)

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Relationships Between Markers of Iron Status and Hematological Parameters in Patients With Sickle Cell Disease.

• Authors : Parrow, Nermi L.¹ (AUTHOR) ; Doherty, Jason M.² (AUTHOR); Conrey, Anna³ (AUTHOR)

• Source: Advances in Hematology. 12/3/2024, Vol. 2024, p1-9. 9p.

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Assessment of total and unbound cell-free heme in plasma of patients with sickle cell disease.

• Authors : Larkin, Sandra; Vichinsky, Elliott; Kuypers, Frans

Subjects: Sickle cell disease; heme; hemolysis

• Source: Experimental Biology and Medicine. 248(10)

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Diverse Approaches to Gene Therapy of Sickle Cell Disease.

• Authors : White, Shanna; Hart, Kevyn; Kohn, Donald

Subjects: CRISPR; gene editing; gene therapy

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Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB‐206 study

• Authors : Kanter, Julie; Thompson, Alexis A; Pierciey, Francis J

Subjects: Medical Biotechnology; Biomedical and Clinical Sciences; Genetics

• Source: American Journal of Hematology. 98(1)

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Design and Implementation of a Sickle Cell Disease Electronic Registry in Resource Limited Setting in Nigeria—A Pilot Study.

• Authors : Idris, Muhammad Aminu¹ (AUTHOR) ; Ruggieri, Lucia² (AUTHOR) ; Ahmad, Hafsat Rufai³ (AUTHOR)

• Source: Hemato. Sep2024, Vol. 5 Issue 3, p340-349. 10p.

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Model‐informed drug development of voxelotor in sickle cell disease: Exposure‐response analysis to support dosing and confirm mechanism of action

• Authors : Green, Michelle L; Savic, Radojka M; Tonda, Margaret

Subjects: Biomedical and Clinical Sciences; Clinical Sciences; Rare Diseases

• Source: CPT Pharmacometrics & Systems Pharmacology. 11(6)

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