rare diseases

accelerated
Biotech

FDA aims for September approval decision for Stealth's drug

The newfound urgency is likely fueled by a public outcry surrounding the FDA's several delays and subsequent refusal of the rare disease candidate.
Gabrielle Masson Aug 21, 2025 10:15am
space launch rocketship

Rocket's gene therapy free from FDA hold

Aug 20, 2025 10:40am
end of the road road block shut down closed blocked

FDA snubs PTC's Friedreich's ataxia hopeful with CRL

Aug 19, 2025 10:55am
clock delay wait time alarm

Regenxbio's Hunter gene therapy latest to face FDA delay

Aug 19, 2025 10:30am
Start Again written on a chalkboard on wooden table

Stealth Bio submits rare disease application for the third time

Aug 18, 2025 11:08am
question ask wonder who why

FDA denies request to reconsider Stealth's ultrarare disease med

Aug 8, 2025 1:58pm