rare diseases

bridge

Biotech

Can’t plausible mechanism principles apply to uniQure?

The framework has a high regulatory bar that could make development in certain diseases more challenging than others, an FDA official told Fierce.

Angus Liu Apr 15, 2026 4:00am

positive negative up down good bad mixed

AstraZeneca’s Strensiq successor misses ph. 3 rare disease goal

Mar 31, 2026 5:35am

Orange flag on top of a mountain

Fierce Pharma

FDA approves Denali's Hunter syndrome drug

Mar 25, 2026 2:50pm

dominoes stop stall halt end red

Genentech drug fails rare disease study, raising obesity questions

Mar 20, 2026 2:11pm

MPS Families Carry Coffin to FDA

Rare disease advocates stage 'funeral' at FDA, make 4 demands

Mar 18, 2026 5:25pm

negative time concept

Stoke aims to restore developmental loss in Dravet syndrome

Mar 18, 2026 2:57pm