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.

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 ...

A new bone marrow transplant method can help cure sickle cell disease and is more accessible than highly expensive gene ...

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

Beam Therapeutics reports Phase 1/2 trial data showing BEAM-302's potential in AATD treatment, with dose-dependent mutation correction and sustained effects.

Recent breakthroughs and regulatory shifts in gene editing might be the key to moving from treating the symptoms of diseases ...

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

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