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.

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.

SCD patients free of VOEs after treatment with gene-editing therapy. Nearly all the patients with severe sickle cell disease (SCD) who were treated with renizgamglogene autogedtemcel (reni-cel) remained free of vaso-occlusive events (VOEs) for up to two years, new data from the Phase 1/2/3 RUBY clinical trial shows.

Sickle cell anemia is an inherited blood disorder. It is caused by a mutation in the gene encoding for hemoglobin, the protein in red blood cells that binds to oxygen so it can be transported throughout the body. The abnormal hemoglobin causes disc-shaped red blood cells to deform into a crescent- or sickle-like shape, causing them to become easily trapped in …

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.. …

Dec 19, 2024 · The treatment also increased total hemoglobin levels and was well tolerated, according to a company press release from Editas Medicine, the developer of the gene-editing therapy.. Data from 28 patients was presented at the Annual Meeting and Exposition of the American Society of Hematology (ASH) in a poster presentation titled, “Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine ...

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. . …

Sep 16, 2024 · These activities help to build a sense of community among people with sickle cell and to educate the broader public. The stories that are shared help to put a human face on the disease, moving it from simply a clinical diagnosis to a broader understanding of the daily struggles and triumphs of those who live with it.. With all of this in mind, it’s also a time to celebrate the resilience and ...

Feb 20, 2024 · Before 2017, hydroxyurea was the only DMT available to SCD patients. Then, between July 2017 and November 2019, three other DMTs were approved: Endari (L-glutamine), Adakveo (crizanlizumab), and Oxbryta (voxelotor). Despite the demonstrated benefits of DMTs in reducing VOCs and improving patient outcomes, studies suggest they are underused, even in patients with a high disease burden.