The investigational gene-editing therapy OTQ923 resulted in normalization of the speed of blood flow in the brain of three adults with sickle cell disease (SCD), according to data from a Phase 1 clinical trial. Findings suggest the treatment could help lower the risk of stroke in patients. The…
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The first healthy volunteer has been dosed in a Phase 1 clinical study of CLY-124 — a novel oral small molecule that Cellarity is developing to treat sickle cell disease — following clearance of the company’s investigational new drug application by the U.S. Food and Drug Administration. “Initiation…
Being a caregiver to someone with sickle cell disease isn’t a role anyone can truly prepare for. It’s a calling that finds us, and it stems from love, loyalty, and necessity. My caregiving journey began with caring for my cousin, a vibrant, witty, and spirited soul whose strength…
Higher pain scores at the time of discharge from the emergency department (ED) may predict the likelihood of hospitalization in children with sickle cell disease (SCD) who seek emergency care for acute pain episodes, according to a study in the U.S. The study also found that patients with severe…
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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