Research map: Addressing the slow uptake of gene therapy for sickle cell disease: a lack of interest, a high-cost, or a more complex issue?
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Papers in this map
Mortality In Sickle Cell Disease -- Life Expectancy and Risk Factors for Early Death
· Orah S. Platt · 1994 · 3373 citations · Cited by this paper
Polycythemia vera and metabolic syndrome: is the relationship between the two diseases more than we think?
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A Review of Disparities in Autologous Stem Cell Transplantation in Patients with Relapsed/Refractory Diffuse Large B-cell Lymphoma
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Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation
· Éliane Gluckman · 2016 · 498 citations · Cited by this paper
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The number of people with sickle‐cell disease in the United States: national and state estimates
· David C. Brousseau · 2009 · 392 citations · Cited by this paper
Reconsidering time from diagnosis to treatment in acute myeloid leukemia: from therapeutic urgency to biologically informed timing
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Maladaptive coping, adaptive coping, and depressive symptoms: Variations across age and depressive state
· Renee J. Thompson · 2010 · 254 citations · Cited by this paper
Apparent plateaus during iron chelation in transfusion-dependent β-thalassemia: interpretation and clinical implications
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Reducing Health Care Disparities in Sickle Cell Disease: A Review
· LaTasha Lee · 2019 · 247 citations · Cited by this paper
Real-world treatment utilization patterns, discontinuation and healthcare resource utilization of first-line Bruton tyrosine kinase inhibitors among elderly patients ≥65 years in chronic lymphocytic leukemia
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Health‐related quality of life in children with sickle cell disease: child and parent perception
· Julie Ann Panepinto · 2005 · 190 citations · Cited by this paper
American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation
· Julie Kanter · 2021 · 139 citations · Cited by this paper
Decreased median survival of adults with sickle cell disease after adjusting for left truncation bias: a pooled analysis
· Michael Rutledge DeBaun · 2018 · 120 citations · Cited by this paper
Navigating the Unknown: Shared Decision-Making in the Face of Uncertainty
· Zackary Dov Berger · 2014 · 98 citations · Cited by this paper
Gene therapy for sickle cell disease: moving from the bench to the bedside
· Allistair Abraham · 2021 · 94 citations · Cited by this paper
Validity of the Child Health Questionnaire for Use In Children With Sickle Cell Disease
· Julie Ann Panepinto · 2004 · 58 citations · Cited by this paper
Bone Marrow Transplantation in Sickle Cell Anemia — The Dilemma of Choice
· Orah S. Platt · 1996 · 48 citations · Cited by this paper
A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease
· Patrick C. DeMartino · 2021 · 42 citations · Cited by this paper
Birth Prevalence of Sickle Cell Disease and County-Level Social Vulnerability — Sickle Cell Data Collection Program, 11 States, 2016–2020
· Mariam Kayle · 2024 · 41 citations · Cited by this paper
How I treat sickle cell disease with hematopoietic cell transplantation
· Elizabeth O. Stenger · 2019 · 35 citations · Cited by this paper
Affordable Pricing of CRISPR Treatments is a Pressing Ethical Imperative
· Jon Rueda · 2024 · 31 citations · Cited by this paper
H3Africa: a model for implementing biobank-based genomic research in resource-constrained settings
· Dhriti Sengupta · 2025 · 27 citations · Cited by this paper
Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance
· Elizabeth O. Stenger · 2022 · 21 citations · Cited by this paper
Estimating Sickle Cell Disease Prevalence By State: A Model Using US-Born and Foreign-Born State-Specific Population Data
· Yao Shi Fu · 2023 · 15 citations · Cited by this paper
Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease
· Mark C. Walters · 2024 · 15 citations · Cited by this paper
Transfusions, disease-modifying treatments, and curative therapies for sickle cell anemia in Africa: where are we now?
· Isaac Odame · 2024 · 15 citations · Cited by this paper
Current state of gene therapy in sickle cell disease
· Mei San Tang · 2024 · 14 citations · Cited by this paper
Dismantling cost and infrastructure barriers to equitable access to gene therapies for sickle cell disease
· Akshay Sharma · 2024 · 14 citations · Cited by this paper
Reforming Reimbursement for the US Food and Drug Administration’s Accelerated Approval Program to Support State Medicaid Programs
· Rachel E. Sachs · 2022 · 13 citations · Cited by this paper
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