rare diseases

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Biotech

Rocket aborts mission to get rare disease gene therapy approved

Having already altered the journey of its rare disease gene therapy toward FDA approval, Rocket Pharmaceuticals has now aborted the mission entirely.
James Waldron Oct 3, 2025 9:30am
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FDA snubs Fortress' Zydus-partnered Menkes disease candidate

Oct 1, 2025 12:00pm
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Applied Tx's stock drops on non-update for rare disease asset

Sep 29, 2025 10:57am
Crossing crosswalk walking road

Ionis rare disease candidate ekes out phase 3 win

Sep 22, 2025 10:46am
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Favicon Fierce Pharma

Stealth scores FDA approval for 1st Barth syndrome treatment

Sep 19, 2025 6:00pm
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Regeneron's ultrarare disease phase 3 hits, teeing up filings

Sep 17, 2025 9:31am