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2025年1月15日 · Orna, Vertex partner on gene-editing therapies for SCD, TDT. Orna Therapeutics is partnering with Vertex Pharmaceuticals to develop gene-editing therapies for …
Vaso-Occlusive Crisis - Sickle Cell Disease News
2022年1月4日 · 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. …
News Archives - Sickle Cell Disease News
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 …
Stem cell transplant may be feasible for young SCD patients
3 天之前 · 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 …
Gates Foundation to invest in Tessera SCD gene-editing treatment
2025年1月9日 · “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 …
Most patients free of vaso-occlusive crises for years after Casgevy
2024年12月12日 · Vertex Pharmaceuticals, which developed Casgevy alongside CRISPR Therapeutics, shared the findings at the 66 th American Society of Hematology (ASH) Annual …
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 …
Celebrating a milestone in gene therapy for sickle cell disease
2024年11月18日 · 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 …
Oxygen therapy for sickle cell disease
2024年8月20日 · What is oxygen therapy? Oxygen therapy, which involves having a person breathe in extra oxygen, is a medical intervention that may be used to help manage acute …
Important things you should know about sickle cell disease
2025年1月8日 · Complications vary from person to person. While numerous potential complications are associated with sickle cell disease, it’s important to note that not every …