The Food and Drug Administration (FDA) announced a new proposal Monday for flexible drug approval pathway treatments ...

Rare diseases are complex medical disorders that are notoriously difficult to diagnose because many present with a wide variety of symptoms that can overlap with more common illnesses. Currently, ...

Even when promising clinical trials exist, and the FDA allows expanded access, rare disease patients like my granddaughter ...

In the five years since Italy-based Chiesi Group established its rare disease division in Boston, the unit has landed regulatory approvals worldwide for 10 therapies — all small molecules and ...

A new AI tool from China called DeepRare is now better than doctors at finding rare diseases. It is correct almost 79% of the ...

Patients who have a particular genetic disease whose symptoms include progressively worsening muscle control were able to walk faster following treatment with an experimental Ionis Pharmaceuticals ...

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare diseases.

Worm tracks show how worms move over time and are the basis for recognising the effects of disease mutations as well as candidate treatments that make disease model worms move like wild type ones. The ...

By Tarun Sai Lomte A new AI-driven diagnostic framework combines clinical, genetic, and phenotypic data to help shorten the ...

Becky Quick leads CNBC Cures, bringing a deeply personal perspective to the multi-media initiative Weekly newsletter, videocast, and website share impactful stories and breaking news around the 30 ...