Advancements in Sickle Cell Disease Treatments and Challenges in Care

The ASH 65th annual meeting saw FDA approvals for two gene therapies for sickle cell disease. Companies like Vertex Pharmaceuticals and Bluebird bio presented updated data for their treatments, which will be available in the U.S. for people with sickle cell aged 12 years or older. A study by Fabian Mueller suggests that CAR-T cell therapy may be effective in treating autoimmune diseases like lupus. A study by Alissa Visram highlights a gap between clinical trial outcomes and real-world effectiveness for multiple myeloma drug regimens. A social media analysis indicates ongoing challenges in caring for people with sickle cell disease.