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University Hospital, Rouen — PHASE3
St. Jude Children's Research Hospital
Emory University — PHASE4
University Hospital, Tours — NA
Fulcrum Therapeutics — PHASE2
Assistance Publique - Hôpitaux de Paris — PHASE1, PHASE2
Mayo Clinic — NA
CSL Behring — PHASE2
Sanofi — PHASE3
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
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Foundation grants
Copay cards
Travel grants
Foundation Grants7
The Assistance Fund — Sickle cell disease
The Assistance Fund
Patient Services Inc — Sickle cell disease
Patient Services Inc
Good Days — Sickle cell disease
Good Days
HealthWell Foundation — Sickle cell disease
HealthWell Foundation
NORD Patient Assistance — Sickle cell disease
NORD Patient Assistance
PAN Foundation — Sickle cell disease
PAN Foundation
Patient Advocate Foundation — Sickle cell disease
Patient Advocate Foundation
Copay Assistance3
Patient Assistance Programs4
Travel Grants12
Bristol Myers Squibb Patient Assistance
Bristol-Myers Squibb
Bristol Myers Squibb Patient Assistance Foundation Application
Bristol Myers Squibb Patient Assistance Foundation
Bristol Myers Squibb Patient Assistance Program
Bristol-Myers Squibb
Bristol-Myers Squibb Patient Assistance Program Application Form
Bristol-Myers Squibb
Emmaus Life Sciences Commercial Co-Payment Assistance Program for Endari
Emmaus Life Sciences
HealthWell: Sickle Cell Disease Financial Support
HealthWell Foundation
Maryland Sickle Cell Disease Association Transportation Assistance Program
Maryland Sickle Cell Disease Association
PAN: Sickle Cell Disease (Medicare)
Patient Access Network Foundation
Sickle Cell Disease Financial Assistance Program
The Assistance Fund
Super T's Mast Cell Foundation Patient Support Grants
Super T's Mast Cell Foundation
The Assistance Fund - Sickle Cell Disease (SCD) Financial Assistance
The Assistance Fund (TAF) Sickle Cell Disease Financial Assistance Program
The Assistance Fund
Lyfgenia
(LOVOTIBEGLOGENE AUTOTEMCEL)Orphan drugGenetix Biotherapeutics Inc.
CASGEVY
(EXAGAMGLOGENE AUTOTEMCEL)Orphan drugVertex Pharmaceuticals Incorporated
ADAKVEO
(CRIZANLIZUMAB)Orphan drugNovartis Pharmaceuticals Corporation
DROXIA
(HYDROXYUREA)Orphan drugWaylis Therapeutics LLC
Endari
(GLUTAMINE)Orphan drugEmmaus Medical, Inc.
OXBRYTA
(VOXELOTOR)Orphan drugGlobal Blood Therapeutics, Inc, A subsidiary of Pfizer Inc.
FERRIPROX
(DEFERIPRONE)Orphan drugstandardChiesi USA, Inc.
Clinical Practice Patterns for Discussing Hydroxyurea Initiation With Families of Children With Sickle Cell Disease.
Despite robust evidence of safety and efficacy, hydroxyurea (HU) uptake remains low for children with sickle cell disease (SCD). Guidelines recommend use of shared decision-making for HU initiation, b...
Effectiveness of preimplantation genetic testing in sickle cell disease: insights from a single-center experience.
Sickle-cell disease (SCD) is a severe autosomal recessive disorder. At-risk couples may prevent transmission either through prenatal diagnosis with possible termination of pregnancy or preimplantation...
Gene Therapy for Sickle Cell Disease.
BackgroundSickle cell disease (SCD) is a genetic blood disorder in which sickled red blood cells cannot easily pass through the vasculature, leading to impaired tissue perfusion and oxygenation that c...
Hemoglobin as a Molecular Glue: Toward Potent Inhibition of HbS Polymerization in Sickle Cell Disease.
Sickle cell disease (SCD), a monogenic disorder arising from a single point mutation in the β-globin gene, continues to pose a significant global health burden despite advances in supportive car...
Myeloperoxidase in the pathogenesis of sickle cell disease.
Sickle cell disease (SCD) is an inherited disorder characterized by abnormal hemoglobin molecules that cause the sickling of red blood cells (RBCs) which occlude blood vessels, increase hemolysis, and...
Benchmarking clonal hematopoiesis in sickle cell disease.
Benchmarking clonal hematopoiesis in sickle cell disease.
Ann Haight, MD, CNA
Children's Healthcare of Atlanta/Emory
📍 TWIN FALLS, ID
Theo Heller, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
📍 Bethesda, Maryland
Leshana St. Jean, PhD
Vanderbilt University Medical Center
Swee Lay Thein, M.D., MD
National Heart, Lung, and Blood Institute (NHLBI)
📍 BETHESDA, MD
Jane Hankins, MD, MS, MD
St. Jude Children's Research Hospital
📍 MEMPHIS, TN
Daniel S Tsze, MD, MPH, MD, MPH
Columbia University
📍 NEW YORK, NY