The U.S. Food and Drug Administration is reviewing an application from Vertex Pharmaceuticals seeking approval of Casgevy (exagamglogene autotemcel) for children with sickle cell disease (SCD) as young as 5 years old. The gene-editing therapy is currently approved in the U.S. and elsewhere for people ages 12 and older with…
Discussion
Last in a series. Read part one. As the sickle cell pain crisis crashed through my body, I started gathering everything I needed before eventually becoming bedbound: water, medication, a heating pad, food. In doing so, I realized I’d become a bit complacent. Because I’d been in such…
A patient with severe sickle cell disease (SCD) remained free of two key SCD symptoms — including painful vaso-occlusive crises — for more than a year after receiving a single dose of CS-206, Correctsequence Therapeutics‘ investigational gene-editing therapy, new data show. The woman represents the first patient…
Discussion
“Why do you have to write about your health on social media?” My brother asked me that question many years ago in our family WhatsApp group after I started openly discussing my sickle cell disease (SCD) online. At the time, I understood his concern. Many families still associate SCD…
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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