Programs

Presentations and Publications

Recent Presentations and Publications | Voxelotor Safety and Efficacy | Voxelotor Mechanism of Action | Selected Key Sickle Cell Disease ResearchSelected Key Transcranial Doppler ResearchAdditional Sickle Cell Disease Research



Voxelotor Safety and Efficacy

Impact of Voxelotor (GBT440) on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease: A Patient Case Report
Hematology Reports, Telfer et al, May 2018

Case Series of Patients with Severe Sickle Cell Disease Treated with Voxelotor (GBT440) by Compassionate Access
American Journal of Hematology, Blyden et al, May 2018

Novel Trial Design to Evaluate Oral Voxelotor for the Treatment of Sickle Cell Disease: Protocol of the Phase 3 Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization (HOPE) Trial (GBT440-031)
American Society of Pediatric Hematology/Oncology Conference 2018, Hoppe et al, May 2018

Initial Results from a Cohort in a Phase 2a Study (GBT440-007) Evaluating Adolescents with Sickle Cell Disease Treated with Multiple Doses of GBT440, a HbS Polymerization Inhibitor
59th American Society of Hematology Annual Meeting & Exposition, Hoppe et al, December 2017

A Single Center Experience of GBT440 Treatment of Severe Anemia in Sickle Cell Disease (SCD)
59th American Society of Hematology Annual Meeting & Exposition, Bridges et al, December 2017

The Pharmacokinetics (PK) of GBT440 Following Single Doses in Pediatric Patients with Sickle Cell Disease (SCD)
59th American Society of Hematology Annual Meeting & Exposition, Washington et al, December 2017

Profound Clinical Benefit in a Patient with Severe, Transfusion-refractory Anemia Treated with GBT440 through Compassionate Use
SCDAA 45th Annual National Convention, Bronte L., October 2017

Efficacy of GBT440 in a Patient with HbSC Genotype Sickle Cell Disease
Academy for Sickle Cell and Thalassemia 11th Annual Conference, Telfer et al, October 2017

The Pharmacokinetics (PK) of GBT440 Are Similar in Adolescents and Adults with Sickle Cell Disease (SCD)
22nd Congress of the European Hematology Association, Washington et al, June 2017

Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor
58th American Society of Hematology Annual Meeting & Exposition, Howard et al, December 2016

Absorption, Metabolism, and Excretion of GBT440, a Novel Hemoglobin S (HbS) Polymerization Inhibitor for the Treatment of Sickle Cell Disease (SCD), in Healthy Male Subjects
58th American Society of Hematology Annual Meeting & Exposition, Rademacher et al, December 2016

GBT440, a Novel HbS Polymerization Inhibitor, Increases Hb Oxygen Affinity and Results in a Rapid Improvement in Hemolysis and Anemia
22nd Congress of the European Hematology Association, Lehrer-Graiwer et al, June 2016

Pharmacokinetics (PK) and Pharmacodynamics (PD) of GBT440, a Novel Hemoglobin S (HbS) Polymerization Inhibitor for the Treatment of Sickle Cell Disease (SCD), in Healthy Volunteers and SCD Patients
22nd Congress of the European Hematology Association, Patel et al, June 2016

GBT440, a novel HbS polymerization inhibitor, increases Hb oxygen affinity and results in a rapid improvement in hemolysis and anemia
10th Sickle Cell Disease Research and Educational Symposium, Lehrer-Graiwer et al, April 2016

GBT440, a Potent Anti-Sickling Hemoglobin Modifier Reduces Hemolysis, Improves Anemia and Nearly Eliminates Sickle Cells in Peripheral Blood of Patients with Sickle Cell Disease
57th American Society of Hematology Annual Meeting & Exposition, Hemmaway et al, December 2015