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Rare Disease Therapeutics
Pharma
Travere CEO Eric Dube to step down
Bradley Campbell, who led Amicus Therapeutics until its recent $4.8 billion acquisition by BioMarin, will become Travere's new CEO.
Angus Liu
Sep 22, 2026 10:10am
FDA approves Ultragenyx gene therapy after prior rejection
Sep 17, 2026 4:50pm
Alkermes’ Lumryz, from $2.4B Avadel buy, hits phase 3 mark in IH
May 12, 2026 11:45am
FDA to reconsider rejected cell therapy. Could uniQure be next?
May 7, 2026 10:40am
In another win for rare disease, Travere nabs FDA’s first FSGS nod
Apr 13, 2026 6:25pm
Fast-rising UCB scores FDA nod for rare disease drug Kygevvi
Nov 4, 2025 11:36am