Pociredir, an investigational oral therapy for sickle cell disease (SCD), was well tolerated and showed signs of promising activity in a group of patients treated with the 12  mg dose in a Phase 1b clinical trial, its developer Fulcrum Therapeutics announced. The ongoing trial, called PIONEER (NCT05169580), aims to…
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…
BEAM-101, a one-time, gene-edited cell therapy, continues to show durable therapeutic benefits more than one year after treatment in people with sickle cell disease (SCD), according to new data from the Phase 1/2 BEACON trial. The treatment’s developer, Beam Therapeutics, shared data from the first 17 patients dosed…
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