5 days ago · First patient enrolled in Phase 1 trial testing fostamatinib for SCD. 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).

10 hours ago · “[This] agreement means eligible sickle cell disease … patients in England now have access to Casgevy,” Vertex said in a company press release.. The deal comes after a positive decision from the National Institute for Health and Care Excellence, or NICE — an independent organization providing guidance on health and social care in England — that recommended Casgevy’s reimbursement.

Feb 4, 2025 · The Phase 1 study (NCT05904093) is sponsored by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), and will be conducted at a single site in Bethesda, Maryland. It expects to enroll up to 25 SCD patients, ages 18 to 65, by invitation. Fostamatinib is the active ingredient in Tavalisse, which is approved in the U.S. for adults with treatment ...

5 days ago · With the addition of Casgevy, CHLA is now offering two gene therapies for sickle cell. The hospital has been offering Lyfgenia (lovotibeglogene autotemcel), a gene therapy that works by providing patients with a gene that encodes a sickle-resistant version of adult hemoglobin.. Both therapies were approved by the U.S. Food and Drug Administration (FDA) in late 2023 to treat people with SCD ...

Jan 23, 2025 · 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 ...

Jan 8, 2025 · Complications vary from person to person. While numerous potential complications are associated with sickle cell disease, it’s important to note that not every patient will experience the same issues. Just because one patient suffers a stroke, for example, doesn’t mean others will face the same fate, even if they have identical genotypes.. …

Jan 9, 2025 · “We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for sickle cell disease worldwide,” Michael Severino, MD, Tessera’s CEO, said in a company press release.. SCD is caused by mutations in a gene that provides instructions for making hemoglobin, the protein that red blood cells use to carry oxygen throughout the body.

Dec 5, 2024 · Therapy provides healthy version of mutated gene. The idea behind gene therapy for SCD and beta thalassemia is to deliver a healthy version of the mutated gene to blood stem cells in the bone marrow, restoring the body’s ability …

Nov 18, 2024 · The extensive research, funding, and other resources required for gene therapy to be approved and for a patient to complete treatment can’t be understated.It also takes extreme courage and patience to be the first at anything in medicine. I commend Cromer and his family for taking this leap. Hopefully a long line of cured patients will follow.

Nov 12, 2024 · Particularly, the study by scientists with Golisano Children’s Hospital will compare sickle cell treatments that help to control disease symptoms, such as blood transfusions and hydroxyurea, with those aiming for a cure, like bone marrow transplantation.. The project aims to recruit nearly 500 children and young adults with SCD, who either underwent a transplant or are using disease ...