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rare diseases

FDA
Biotech

At CNPV hearing, industry support meets ‘concern’ from experts

Comments at the June 4 public hearing largely fell along three main fault lines.
Darren Incorvaia Jun 5, 2026 5:00am
Three blocks displaying from left to right a person a handshake and another person

Servier inks $2.6B buyout of Edgewise’s muscular dystrophy unit

Jun 1, 2026 8:31am
success failure signposts

AstraZeneca, after phase 3 miss, posts data behind approval bid

May 29, 2026 10:15am
delay

FDA extends Beren’s rare disease review after info request

May 28, 2026 10:45am
A nostalgic steam train travels through a snowy winter forest in Geesthacht Germany

Relay ‘full steam ahead’ on PI3K drug after rare disease readout

May 19, 2026 6:00am
Lung

Sanofi, Wave post updates in race to end AATD’s 40-year drought

May 18, 2026 4:30pm

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