pediatric rare disease

Durham, North Carolina — September 22, 2026 Ophirex announced publication of results from the Phase 2 BRAVIO clinical trial...

Shanghai, China, September 21, 2026 Abbisko Therapeutics has announced positive preliminary results from a Phase 2 clinical study evaluating...

Boston, Massachusetts, August 5, 2026 Galibra Neuroscience has received both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation...

Brooklyn, New York, August 5, 2026 Phoenix Nest Inc. has been awarded a $2,984,658 Small Business Innovation Research (SBIR)...

PALO ALTO, Calif., June 28, 2026 BridgeBio Pharma announced that positive results from its pivotal Phase 3 PROPEL 3...

PARMA, Italy, June 5, 2026 Chiesi Global Rare Diseases has announced a major regulatory milestone following the European Commission’s...

NASHVILLE, Tenn., Feb. 4, 2026 —Cumberland Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has granted Fast...

NEEDHAM, Mass., September 19, 2025 – Stealth BioTherapeutics Inc. today announced that the U.S. Food and Drug Administration (FDA)...