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

The PHOENIX study is a 24-month, randomized, double-masked, placebo-controlled, multicenter, pivotal Phase 3 trial. The study is ongoing across sites in the United States, the United Kingdom, France, ...

Minovia Therapeutics said Monday that the Food and Drug Administration granted fast-track and rare-pediatric-disease designations to its lead investigational compound. The drug, MNV-201, is in Phase 2 ...

Shares of Precision BioSciences, Inc. (NASDAQ:DTIL) jumped 17% on Wednesday after the company announced that its gene therapy ...

Precision BioSciences (NASDAQ:DTIL)  announced Wednesday that its experimental therapy PBGENE-DMD has received Rare Pediatric ...

Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD).

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 ...

Sanofi and Regeneron Pharmaceuticals said they got Food and Drug Administration approval for anti-inflammatory drug Dupixent as a treatment for a rare skin disease, adding an eighth indication in ...

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 ...

In 2009, the account executive was diagnosed with Advanced IgA nephropathy, a rare kidney disease. Last year, he got the news he dreaded. At 39 years old, he needed a new kidney.

Terminally-ill B.C. girl with extremely rare disease will no longer have drug funded By Richard Zussman & Amy Judd Global News Posted June 18, 2025 3:13 pm Updated June 19, 2025 5:46 pm ...