For nearly half of people with sickle cell disease who took part in the Phase 2 part of a clinical trial called RISE UP, treatment with the oral medication mitapivat brought about a significant increase in levels of hemoglobin — the oxygen-carrying protein in red blood cells — new…
News
The Sickle Cell Disease Association of America (SCDAA) and the MedicAlert Foundation are partnering on a pilot program to help those living with sickle cell disease (SCD) receive faster and better emergency care. With the aim of improving the safety and well-being of patients during acute pain…
A digital portrait created by artist Nikkolas Smith for the American Red Cross seeks to heighten awareness of sickle cell disease (SCD) and the ongoing need for blood donations for SCD patients, particularly from Black donors. The artwork, titled “Transfusion,” depicts a young African American person crossing their hands,…
The global burden of sickle cell disease (SCD) may be greater than previously thought, with mortality rates up to 11 times higher than earlier estimates, a recent analysis reported. Particularly vulnerable are people living in sub-Saharan Africa and children younger than age 5. Failures to properly diagnose sickle cell…
The U.S. Food and Drug Administration (FDA) is expected to decide before year’s end on whether or not to approve the experimental gene therapy lovotibeglogene autotemcel — known as lovo-cel — for people with sickle cell disease (SCD) who have a history of painful vaso-occlusive crises (VOCs). The…
Children’s of Alabama hosted a special event, in collaboration with famed football coach Nick Saban, to deliver a flock of social robot ducks — the My Special Aflac Duck, free to certain pediatric patients — to 18 children in the state with sickle cell disease (SCD) and cancer.
With events ranging from a candlelight vigil to a new hospital exhibit to a 5K, supporters are poised to mark World Sickle Cell Day on June 19 to heighten awareness of the genetic blood disorder that affects millions of people internationally — and about 100,000 individuals in the U.S. In…
The U.S. Food and Drug Administration (FDA) is reviewing an application that seeks the approval of exagamglogene autotemcel, a CRISPR-based gene-editing therapy known as exa-cel, to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The rolling biologics license application was completed by Vertex Pharmaceuticals and CRISPR…
A protein called NLRP12 appears to act as a switch that turns on inflammation and tissue damage when it senses free heme, which is released from broken red blood cells during an infection and in other diseases like sickle cell disease (SCD). This protein could therefore be a potential…
The Committee for Medicinal Products for Human Use (CHMP) is recommending the conditional approval of Adakveo (crizanlizumab) for sickle cell disease (SCD) be revoked in Europe. According to the therapy’s developer Novartis, a final decision from the European Commission is expected in about two months. In the…
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