Karl Fitzpatrick has described his frightening ordeal after being diagnosed with potentially life-threatening Guillain–Barré ...

Both events are taking place simultaneously 19–20 November in Brussels, Belgium. As interest in rare disease drug development ...

The FDA has cleared PTC Ther­a­peu­tics’ gene ther­a­py for AADC de­fi­cien­cy, an ul­tra-rare dis­ease that im­pacts the ...

to use its artificial intelligence to identify novel rare disease indications for a discontinued drug asset. Under the terms of the agreement, Sanofi will provide data related to the unnamed drug ...

Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease ...

Recruiting patients for rare disease trials is akin to finding a needle in a haystack, with challenges such as geographical ...

Until now, support for the diagnosis and treatment of rare diseases has been limited to drug producers and sellers, but the ...

By Dr. Kaushik Mandal Rare diseases very often remain in the shadows, as common people have limited awareness about them.

In Tulsa, mechanic Matt Parker rallied the community to support 21-year-old Corbin Norcom, who has a fatal disease, by ...

The increased RM25 million allocation in the 2025 Budget for rare disease treatment costs is expected to improve haemophilia ...

Profit after tax came in ₹38.4 crore in Q2 of FY25, a recovery from a net loss of ₹11.8 crore in the first quarter of this ...

An Ottawa boy with a rare disease will be getting the chance to see Taylor Swift in concert after the winner and organizers ...