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 ...

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

Ms Siti Nur Sabrina Anis, 26, had to swop one set of wheels for another – her Yamaha motorcycle for a motorised wheelchair.

Across 100 patients in three Phase III studies, Vertex's Casgevy showed lasting benefits for more than five years.

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

One-time surgical application of genetically engineered autologous keratinocyte sheets provides significant closure and pain ...

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

Suki Tipp was diagnosed with indolent systemic mastocytosis, a rare blood disorder that triggers unpredictable and ...

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 ...