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.
With spring break quickly approaching, let’s take a look at some breaking scientific discoveries and news, from a new sickle ...
The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...
ROCHESTER, N.Y. – Researchers at Golisano Children’s Hospital have received a multi-million-dollar grant to lead a ...
Beam Therapeutics reports Phase 1/2 trial data showing BEAM-302's potential in AATD treatment, with dose-dependent mutation correction and sustained effects.
Explore the benefits of adopting universal molecular genetic testing for newborn screening of haemoglobinopathies to improve ...
Recent headlines in the U.K. lauded the approval of a new sickle cell cure using gene-editing technology. Columnist Mary Shaniqua has doubts.
Still, Novo is steadfast in its goal to eventually become a leader in the space, the company’s EVP of rare disease, Ludovic ...
Vertex Pharmaceuticals operates in a profitable subsector and is poised for further success, says Dr Michael Tubbs ...
Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple ...
When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters ...
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