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…
The first patient with severe sickle cell disease (SCD) who received a single infusion of CTX001, an experimental gene-editing cell therapy, remains free of vaso-occlusive crises (VOCs) nine months after treatment, a Phase 1/2 clinical trial shows. Findings from the trial, “(S280) Initial Safety…
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…
Supporters are poised to mark World Sickle Cell Day, observed each June 19 to raise awareness about the red blood cell disorder, and to raise funds to fight it. Across the globe, supporters will participate in activities such as wearing red for sickle cell disease (SCD), sponsoring a…
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