The FDA granted orphan drug designation to Sineugene Therapeutics' SNUG01, a first-in-class experimental gene therapy for ...

Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial ...

CRISPR technology is transforming modern medicine by enabling precise DNA editing to treat genetic disorders, including sickle cell anemia and beta-thalassemia, experts say.

Gabriella Ngang, of Farmington Hills, is one of three in Michigan to take part in a clinical trial of a new sickle cell ...

Boosting Klotho protein levels in mice improves physical and cognitive aging, offering potential for future longevity ...

The GOP tax bill repeals the Inflation Reduction Act’s price controls for drugs for rare diseases.

New technologies and a proactive mindset are transforming how glaucoma is diagnosed and treated—and redefining what ...

Using an AI-driven drug discovery process, the drug known as vorinostat was identified as a promising treatment for Rett ...

Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are ...

Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are ...

The research reveals how targeting the KMT2D gene could help correct oral disorders and prevent craniofacial birth defects.