A European Medicines Agency (EMA) committee has adopted a positive opinion on Novartis‘s Adakveo (crizanlizumab) for the prevention of vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended the conditional approval of…
Most people with sickle cell disease (SCD) in the U.S. are satisfied with their primary care practitioners, but less so with emergency care given at a hospital to manage severe pain, according to a survey of more than 400 teenage and adult patients. How a person expects to be…
Cannabis could be a safe and potentially effective way of easing chronic pain in people with sickle cell disease (SCD), according to results of a small clinical trial. Although the benefits in pain were not statistically significant compared to a placebo, pain interference in mood decreased over the five-day…
An umbilical cord blood transplant, coupled with a reduced intensity conditioning (RIC) regimen, can safely and effectively treat children with genetic disorders that include sickle cell disease (SCD) and thalassemia, data from a Phase 2 trial show. These findings are detailed in the study, “Reduced-intensity single-unit…
Blood stem cell transplant from unrelated donors is a feasible strategy in people with sickle cell disease (SCD), with better outcomes when blood markers are matched between donor and recipient, a study suggests. The study, “The role of HLA matching in unrelated donor hematopoietic stem cell…
Bronchodilators used to treat asthma could worsen sickle cell disease (SCD) in children and teens, and contribute to a greater risk of vaso-occlusive crisis (VOC), a study suggests. The study, “Salbutamol Worsens the Autonomic Nervous System Dysfunction of Children With Sickle Cell Disease”…
FT-4202 — an investigational treatment for sickle cell disease (SCD) — has a favorable safety profile and the expected biological effects, providing preliminary support of efficacy, according to data from an ongoing Phase 1 clinical trial. Forma Therapeutics, which is developing FT-4202, plans to start a global Phase…
While it will be entirely virtual this year, the 7th Annual Walk With the Stars & Move-A-Thon is taking place July 11 to raise awareness and money to fight sickle cell disease (SCD). Presented by the Sickle Cell Disease Association of America (SCDAA), the signature 5K national event is…
Global Blood Therapeutics (GBT) plans to submit a marketing authorization application to the European Medicines Agency (EMA) asking that Oxbryta (voxelotor) be approved for treating hemolytic anemia in people with sickle cell disease (SCD) who are 12 or older. The submission is anticipated by…
Mitapivat (AG-348) safely increased the amount of hemoglobin and changed the levels of biomarkers of red blood damage and energy production in adults with sickle cell disease (SCD), early Phase 1 trial data show. “These data build on our six years of clinical experience with this mechanism…
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