The U.S. Food and Drug Administration (FDA) has granted priority review — a decision that could speed a drug’s approval — to an application for the oral therapy mitapivat for sickle cell disease (SCD). According to developer Agios Pharmaceuticals, mitapivat, if approved, will be the first therapy of…
Discussion
One of the hardest things about growing up with sickle cell disease wasn’t the pain. It was the limitations — not just those imposed by the condition itself, but also from other people’s expectations. Like many patients, I grew up hearing what I couldn’t do. The conversations weren’t always…
Discussion
Casgevy (exagamglogene autotemcel) is now approved in the U.S. to treat children as young as 2 with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) initially approved the Vertex Pharmaceuticals’ gene-editing therapy in 2023 for SCD patients ages 12 and older who…
Discussion
If you’re like me and have a good medical team, over time, you’ve likely grown attached to them. When you’re fortunate enough to have a competent and consistent team that genuinely cares about your health, those types of relationships matter in ways that go beyond doctor appointments. That type of…
Becoming educated and informed about sickle cell disease is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your sickle cell disease journey.
A stem cell transplant is the only potential cure for sickle cell disease, but there are a variety of other treatment approaches. Here you can find more information about current treatments as well as experimental therapies in the pipeline.
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