Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?

Measles is highly contagious among those who are unvaccinated; outbreaks can have severe health consequences in children ...

Senators made the right decision for America's most vulnerable patients ― now, the House must follow suit. The ORPHAN Cures Act is a simple fix to a costly mistake ― and a necessary step to keep rare ...

Tel Aviv University researchers say their investigation into a GRIN2D gene mutation will advance research into a protein ...

R136S, or the “Christchurch mutation,” appears to protect against Alzheimer’s disease by suppressing inflammatory signaling ...

The designation reinforces the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. It follows earlier regulatory recognition by the European Medicines ...

The selected projects demonstrated significant potential to address unmet needs within these rare communities. The recipients include: Fabry disease: Mitra Tavakoli, University of Exeter, Exeter ...

Rare Kidney Disease Foundation spreading awareness on disease by: Bridge Street Staff Posted: Jun 19, 2025 / 11:42 AM EDT Updated: Jun 19, 2025 / 01:37 PM EDT ...

A rare and highly contagious disease has been found at two commercial pigeon-breeding farms in Chilliwack. The Canadian Food Inspection Agency (CFIA) said Newcastle disease was found in samples ...

Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s ...

BioMarin in 2023 disclosed that it received a subpoena from the Justice Department requesting information regarding sponsored testing programs related to two of the company's enzyme replacement ...

Effects of overexpression of FOXG1 WT and variants on neuronal migration by IUE. Credit: Molecular Psychiatry (2025). DOI: 10.1038/s41380-025-03077-y ...