Guest Voice: Moving, literally, in the right direction for sickle cell care. Richard Bayliss is 44 years old and has sickle cell disease with hemoglobin SS disease.

22 hours 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 ...

2 days ago · 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 ...

Jan 4, 2022 · Sickle cell disease (SCD) is an inherited disorder caused by a mutation in the gene encoding a subunit of hemoglobin, the protein that carries oxygen in red blood cells. Mutant hemoglobin — called sickle hemoglobin or hemoglobin S — tends to clump together in red blood cells, causing them to take on an irregular “sickle-like” shape that gives the disease its name.

Jan 31, 2025 · New treatments and therapies are being developed every day for people with sickle cell disease. I take hydroxycarbamide daily. The medicine was initially designed for cancer patients, but sickle cell patients have also noted improvements in well-being while taking it.

Dec 12, 2024 · Vertex Pharmaceuticals, which developed Casgevy alongside CRISPR Therapeutics, shared the findings at the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 7-10 in San Diego and virtually. The presentation was titled “ Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease.” Data specifically showed that, among 42 patients ...

Jan 28, 2025 · Symptoms of SCD include anemia, fatigue, swelling of the extremities, recurrent infections, and vision problems. Sickled red blood cells can also block blood flow, especially in small blood vessels, leading to painful vaso-occlusive crises and other serious complications.. Stem cell transplants are available, and have the potential to cure SCD. But such treatment carries significant risks and ...

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.. …

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.

Sickle Cell Disease News is a digital platform intended to provide the sickle cell disease (SCD) community with the most recent news and information on SCD, as well as first-hand community perspectives from our patient and caregiver columnists.. All articles on Sickle Cell Disease News are original content produced by in-house writers and editors. . …