Nearly all the patients with severe SCD treated with the gene-editing therapy reni-cel remained free of VOEs for up to two ...

Data from a Phase 2/3 trial shows etavopivat may reduce the incidence of vaso-occlusive crises and raise levels of hemoglobin ...

that her hemoglobin proteins, which carried oxygen through her body, were catching on each other, forming unwieldy chains, ...

The first medical treatment to use Crispr gene editing has been on the market for a year. Its complexity means few patients ...

There are many types of sickle cell disease. The majority of cases are divided into three subtypes depending on which genes you inherited: Hemoglobin is an iron-containing protein found in your ...

Sickle cell disease refers to a group of genetic diseases that impact a protein that ferries oxygen through the blood. That protein is called hemoglobin ... | Genetics And Genomics ...

Transplant is beneficial earlier in life, Wang told MedPage Today, but carries potential short- and long-term side effects.

Eleven-year-old Jayden Randall of Carrick is one of the children who is alive and thriving today thanks to UPMC Children's ...

Base editing gene therapy is rewriting the future for sickle cell patients, leaving them healthier and pain-free.

"The education was not there. It still isn't there, but it should be," Alvin Henry Jr., a sickle cell disease patient, said. ...

European Commission adopts positive opinion on Agios’ Mitapivat for orphan medicinal product designation in sickle cell disease: Cambridge, Mass Saturday, December 21, 2024, 12: ...