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…
Stem cell transplant from a partially matched donor is more cost-effective than gene therapy to treat sickle cell disease (SCD), according to a study. In order for currently available gene therapies to deliver cost effectiveness on par with stem cell transplant, their cost would need to come down…
Fulcrum Therapeutics said it is discontinuing development of pociredir, its experimental oral treatment for sickle cell disease (SCD), after the U.S. Food and Drug Administration (FDA) raised concerns that the therapy may increase the risk of certain blood cancers. The concerns stem from the unexpectedly high rate 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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