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3 days ago · Orna, Vertex partner on gene-editing therapies for SCD, TDT. Orna Therapeutics is partnering with Vertex Pharmaceuticals to develop gene-editing therapies for people with …
Vaso-Occlusive Crisis - Sickle Cell Disease News
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 …
Most patients free of vaso-occlusive crises for years after Casgevy
Dec 12, 2024 · Vertex Pharmaceuticals, which developed Casgevy alongside CRISPR Therapeutics, shared the findings at the 66 th American Society of Hematology (ASH) Annual …
Most sickle cell patients VOE-free after Lyfgenia gene therapy
Dec 12, 2023 · That’s according to new data from Lyfgenia’s developer, Bluebird Bio, at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition.Also known as lovo …
Celebrating a milestone in gene therapy for sickle cell disease
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 …
SCD patients free of VOEs following gene-editing therapy reni-cel
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 …
Study to compare SCD treatments for symptoms vs. a transplant
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 …
Low-intensity stem cell transplants may benefit SCD patients: Study
Sep 3, 2024 · “By using a low-intensity blood stem cell transplant for sickle cell disease, we may be able to stop the cycle of lung injury [seen in SCD patients] and prevent continued damage,” …
About Us – Sickle Cell Disease News
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 …
I’m tired of watching sickle cell patients have preventable deaths
Nov 22, 2024 · Here in the U.K., I’ve seen two recent reports about sickle cell patients dying from negligence. Both of their deaths were preventable. As MyLondon reported in September, Dave …