The FDA has recently approved of an updated label for Endari, a treatment for sickle cell disease (SCD). This new label will give medical professionals better information, allowing them to…
Fulcrum Therapeutics is beginning a Phase 1 trial of FTX-6058, a treatment for sickle cell disease (SCD). Researchers will focus on the tolerability, safety, and pharmacokinetics of the medication. The…
CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…
Elliot Preddie, age 12, is ready to get back to his favorite sport, football. He had to put off playing due to sickle cell disease (SCD). Elliot substituted taekwondo and…
Editas Medicine has developed a sickle cell disease (SCD) treatment, and it has recently received the Rare Pediatric Disease designation from the FDA. This means that their therapy, EDIT-301, is…
By Lauren Thayer from In The Cloud Copy Sickle cell disease (SCD) is group of inherited red blood cell disorders. In a typical, healthy red blood cell, the cell is…
Global Blood Therapeutics (GBT) will submit their Marketing Authorization Application (MAA) for Oxbryta to the EMA by 2021, says a recent BioSpace press release. Ultimately, the MAA will seek regulatory…
June 19th is recognized as World Sickle Cell Day. This day is set aside to help spread awareness about sickle cell disease and its variants among the general public and…
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