Aaron Scott, of Fayetteville, is the first in North Carolina to receive a new FDA-approved therapy which uses the patient's own cells to relieve the pain of sickle cell disease.

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

Explore the benefits of adopting universal molecular genetic testing for newborn screening of haemoglobinopathies to improve ...

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters ...

Learn how CASGEVY, the first NHS-approved CRISPR gene therapy, treats sickle cell disease through a revolutionary genetic ...

ROCHESTER, N.Y. – Researchers at Golisano Children’s Hospital have received a multi-million-dollar grant to lead a ...

Esther Nkemakolam runs her fingers through kinetic sand and sings a “Moana” song as UPMC Children’s Hospital nurse Taylor ...

One of the major contributors to complications in SCD is oxidative stress, a condition where the body’s antioxidant defense ...

Still, Novo is steadfast in its goal to eventually become a leader in the space, the company’s EVP of rare disease, Ludovic ...

HUNTSVILLE, Ala. — A blood disorder that primarily affects minority communities, sickle cell disease changes the shape and ...

Last month, the NICE endorsed Casgevy for use in the NHS under a managed access scheme to treat sickle cell disease.