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

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

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 ...

“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 ...

Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting friends. It was a contrast to how Cole, 18, spent part of the 2024 holiday ...

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 ...

For the first time in his life, a young New Jersey man is pain-free thanks to a new gene treatment working to cure patients of sickle cell disease. Gerald Quartey, 18, is the first patient in the ...

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 ...