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Voxelotor for Sickle Cell Disease Wins FDA Nod for Patients as Young as 4
Voxelotor has a novel mechanism of action. The first-in-class therapy regulates the affinity of hemoglobin for oxygen, resulting in a decrease in the concentration of deoxygenated sickle hemoglobin (HbS), which forms polymers. It’s hypothesized that because oxygenated HbS cannot polymerize, modifying HbS to increase the proportion of oxygenated to deoxygenated HbS in red blood cells would alter disease severity.
The FDA Friday approved the wider use of voxelotor (Oxbryta) in patients with sickle cell disease (SCD) as young as 4 years of age. In 2019, the oral polymerization inhibitor was approved by the FDA for adults and pediatric patients with SCD 12 years of age and older.
people & placesTammuella (Tami) Singleton, MDDr. Singleton serves as a pediatric hema...
education & researchPiracetam for reducing the incidence of painful sickle cell disease crisesBACKGROUND: Sickle cell disease is one ...
education & researchRisk factor analysis of cerebral white matter hyperintensities in children with sickle cell diseaseSickle cell disease (SCD) is complicated...
people & placesJerlym S. Porter, PhD, MPHJerlym S. Porter is an Associate Member ...
news & eventsSickle cell trait may not increase the risk of deathPeople who carry a gene for sickle cell ...
news & eventsNew Pre-transplant Treatment Regimen Improves Survival of Kids with Sickle Cell Disease, Trial ShowsA new pre-transplant conditioning regime...
news & eventsFDA Approves First Targeted Therapy to Treat Patients with Painful Complication of Sickle Cell DiseaseToday, the U.S. Food and Drug Administra...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.