The Gates Foundation will invest up to $50 million to support Tessera's SCD gene editing treatment, the company said.
More than 500,000 babies were tested for sickle cell disease in Catalonia’s newborn screening program, with 160 cases ...
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 ...
The following is a summary of “Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 ...
"Sickle cell disease has a broad spectrum of severity ... instructions for his red blood cells to make a fetal form of hemoglobin. (Unlike "adult" hemoglobin, fetal hemoglobin doesn't cause ...
Gene-editing kicked off 2024 with a bang in the medical sector but closed the year with a whimper. It started with the FDA ...
The new gene therapies for sickle cell disease—including the gene-editing ... that raises fetal hemoglobin levels. The FDA approved it in 1998. But Orkin says hydroxyurea and other available ...
They didn’t know that she carried a genetic blueprint for a misshapen molecule; that her hemoglobin proteins ... newborn’s heel and test it for sickle cell disease. Catch it early and you ...
In sickle cell disease, a gene mutation causes the hemoglobin molecules in red blood cells to form a rigid chain. This causes the cells to stiffen into a sickle shape, clogging blood vessels and ...
“Sickle cell disease (SCD) has a broad spectrum of ... instructions for his red blood cells to make a fetal form of hemoglobin. (Unlike “adult” hemoglobin, fetal hemoglobin doesn’t cause ...