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rare diseases
Pharma
As Pfizer bows out, CSL keeps hemophilia gene therapy hopes alive
A small patient population, a wait-and-see attitude and a “fairly significant” infrastructure barrier have slowed CSL's gene therapy rollout.
Angus Liu
Apr 11, 2025 10:32am
Rhythm preps filings in rare obesity disease after trial win
Apr 7, 2025 9:52am
Amgen's Uplizna expansion push bears fruit with rare disease nod
Apr 4, 2025 11:05am
CBER chief Peter Marks resigns from FDA
Mar 31, 2025 10:50am
FDA approves Soleno's Vykat XR for insatiable hunger disorder
Mar 27, 2025 11:24am
Novartis' Fabhalta nod in C3G leaves room for rival Apellis
Mar 21, 2025 10:37am
