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GBT601 and inclacumab, experimental therapies from Global Blood Therapeutics (GBT) for sickle cell disease (SCD) and associated vaso-occlusive crises (VOCs), were generally safe and showed signs of biological activity in healthy volunteers, according to data from Phase 1 clinical trials. These findings will be presented by GBT through two…
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Long-term treatment with Oxbryta (voxelotor) safely and effectively reduced anemia and red blood cell destruction (hemolysis), while keeping a low rate of painful vaso-occlusive crises (VOCs), in adolescents and adults with sickle cell disease (SCD). That’s according to nearly three years of data from the Phase 3 HOPE…
Through a new partnership between Emmaus Life Sciences and UpScript, patients with sickle cell disease (SCD) will soon be able to consult with a physician from home and have greater access to Endari (L-glutamine). Through telehealth — using computers and mobile devices to manage and receive…
The first participant has been enrolled in the Phase 1/2 clinical trial CEDAR evaluating Graphite Bio’s investigational gene editing therapy GPH101, which is designed to directly correct the genetic mutation that causes sickle cell disease (SCD). “We are thrilled that our first patient is now enrolled in our CEDAR…
The U.S. Food and Drug Administration (FDA) has cleared Beam Therapeutics to start clinical testing of BEAM-101, the company’s investigational gene editing cell therapy for sickle cell disease (SCD). “BEAM-101 has the potential to offer a one-time treatment for patients with sickle cell disease, and this clearance enables…
When a trip to Spain resulted in a hospitalization, columnist Dunstan Nicol-Wilson learned the importance of preparation with sickle cell.
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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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