Pfizer announced on September 25, 2024, that they are voluntarily withdrawing all lots of OXBRYTA (voxelotor) and discontinuing all active clinical trials and expanded access programs worldwide.
Read moreSickle Cell Gene Therapies and CMS Cell and Gene Therapy Access Model
The federal government has created a program called the Cell and Gene Therapy Access Model, the goal of which is to provide people with Medicaid who have rare and severe diseases access to potentially transformative treatments.
Read moreGene Therapy Approved for Transfusion-Dependent Thalassemia
With the approval of Casgevy, there are now two gene therapies, Casgevy and Lyfgenia, for the treatment of transfusion dependent thalassemia (TDT).
Read moreSickle Cell Disease: Hope for the Future
Every June 19, we observe World Sickle Cell Day to raise international awareness of sickle cell disease and the challenges patients and families face when handling the disease. Sickle cell disease is a group of inherited red blood cell (RBC) disorders that affect hemoglobin, the protein that carries oxygen through the body.
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