Sickle cell disease (SCD) is associated with enhanced immune B-cell responses and the production of self-reactive antibodies that target and destroy red blood cells, according to data from a mouse model of the disease and patient samples. These changes in immune responses were regulated by type-1 interferons (IFN-1), an…
A sickle cell disease (SCD) patient in Bahrain received the gene-editing therapy Casgevy (exagamglogene autotemcel), becoming the first patient outside the U.S. to be treated with the drug. Bahrain approved Casgevy in late 2023, becoming the first country in the Middle East to approve the therapy and the…
Some children with sickle cell disease (SCD) may benefit from supplementing with folic acid, according to a clinical trial. Supplementation was found to significantly increase the levels of red blood cell folate, a molecule important for the production of red blood cells, compared with a placebo. However, no differences…
Vertex Pharmaceuticals has entered a reimbursement agreement with the National Health Service in England that will enable eligible sickle cell disease (SCD) patients in that country to get access to treatment with Casgevy (exagamglogene autotemcel) via the public health system. “[This] agreement means eligible sickle cell disease…
Children’s Hospital Los Angeles (CHLA) is now offering Casgevy (exagamglogene autotemcel), an approved gene-editing therapy for sickle cell disease (SCD). “This is a transformative time for Children’s Hospital Los Angeles and the pediatric community we serve,” Alan S. Wayne, MD, pediatrician-in-chief at CHLA, said in a press…
The first patient has been enrolled in a Phase 1 clinical trial evaluating the safety and tolerability of Rigel Pharmaceuticals’ fostamatinib — a medication approved in the U.S. for treating an autoimmune condition — in people with sickle cell disease (SCD). The Phase 1 study (NCT05904093) is…
People with sickle cell disease (SCD) have brains that appear to be more than a decade older than their actual age. That’s according to a recent study that also found that patients with older-looking brains had poorer cognitive function. “Our study explains how a chronic illness … can cause…
Immvention Therapeutix has partnered with Novo Nordisk to develop oral treatments for people with sickle cell disease (SCD) and other chronic conditions. The U.S.-based Immvention called the deal “a strategic collaboration and license agreement” in a company press release. “We are excited to partner with Novo…
A hematopoietic stem cell transplant (HCT) may be a feasible treatment option for adolescents and adults with severe sickle cell disease (SCD), according to a U.S. study. Two-year survival rates were similar among patients who had an available donor for HCT and among those who did not, although they were…
Fulcrum Therapeutics is planning to report, by midyear, new data from the ongoing PIONEER Phase 1b clinical trial that’s testing its oral therapy candidate pociredir (FTX-6058) in people with sickle cell disease (SCD), according to a new corporate presentation. That will include data from the study’s third-highest…
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