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

A new study led by UC San Diego’s Scripps Institution of Oceanography may pave the way for lab-based production of rare coral ...

A researcher says the experimental use of gene therapy for a rare inherited disorder is saving almost as much money for ...

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.

A strategy that analyzes the structural properties of RNA could help identify regions that are promising targets for antiviral drugs.

July kicks off the latter half of 2025, and the first six months of this year have been eventful, to say the least. Yet, the ...

State Medicaid leaders create a plan to ensure safety, affordability, and equitable access for children and families living ...