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 ...

With spring break quickly approaching, let’s take a look at some breaking scientific discoveries and news, from a new sickle ...

Sickle Cell Disease is a complex and debilitating genetic blood disorder that primarily affects people of African, ...

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

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

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

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 ...

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

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