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orphan drug
Biotech
More collaborative FDA can accelerate rare disease R&D: report
The National Academies is recommending the FDA and European Medicines Agency implement a “navigation service” to advise on regulatory pathways.
Nick Paul Taylor
Sep 13, 2024 5:00am
As pharma eyes shiny obesity drugs, Vertex tops orphan drug list
Apr 24, 2024 12:01am
Pfizer punts pair of orphan drugs amid rare disease R&D rethink
Jan 31, 2023 9:15am
Stealth granted FDA meeting to revive elamipretide filing
Jun 14, 2022 10:10am
Selecta clinical hold on gene therapy lifted
Mar 10, 2022 10:44am