The Archduchess, who lived in Nice, leaves behind her husband  Archduke Carl Christian and their five children.

As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.

There are approximately 7,000 different types of rare disease and researchers estimate that there are more than 300 million people worldwide living with such a condition. When taken ...

a New Jersey mother credits an "amazing" new drug for stopping her disease in its tracks. Raziel Green, 52, an active runner and mother of two, was diagnosed with a rare form of ALS in 2017.

A phase 3 trial of AstraZeneca’s $800 million rare disease candidate has met its primary endpoint. The update keeps eneboparatide on track to come to market, but the lack of numbers in the ...

June 10, 2024 — Scientists have grown 'mini-guts' in the lab to help understand Crohn's disease, showing that 'switches' that modify DNA in gut cells play an ... Apr. 24, 2024 — Study ...

Archduchess Estelle of Austria has died at the age of 46, leaving European aristocracy in mourning. The royal passed away ...

The FDA granted the therapy both orphan drug and rare pediatric disease designations in early February. A two-part phase 1 trial of ABO-101 is slated to start this month, according to ...

Chiesi USA of Cary and its European partner Holostem Terapie Avanzate have received Orphan Drug Designation from the U.S. Food and Drug Administration for an investigational cell-based therapy for a ...