CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

“It can hit you out of nowhere,” she tells TODAY.com.

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated ...

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...

While African governments expand basic treatment, high-income countries adopt the first gene-editing therapy to cure sickle cell.

Mean absolute fetal hemoglobin (HbF) increased by 12.2% (from 7.1% to 19.3%) at Week 12 (n=12), representing a rapid, robust, and clinically ...

Recent therapies like hydroxyurea (Droxia, Bristol-Myers Squibb), the first FDA-approved drug for SCD in 1994, increase fetal hemoglobin (HbF), which, through unknown pathways, improves SCD symptoms ...

Sickle cell anemia (HbSS) is the homozygous dominant variant and the most common and severe form of the disease. Whereas patients who only inherited one gene encoding for abnormal HbS and a different ...

UCSF researchers found that 64.4% of female patients report sickle cell disease (SCD) pain during menstruation. However, ...

You Look Fine” is a groundbreaking new documentary, offering an intimate, first-person account of living with sickle cell disease.

This article is part of a Rare Disease series sponsored by DeepIntent. With Rare Disease Month wrapping up and Rare Disease Day taking place on Saturday, MM+M is celebrating a handful of campaigns ...