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orphan drug

Combine
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
pipeline pipes RD research

As pharma eyes shiny obesity drugs, Vertex tops orphan drug list

Apr 24, 2024 12:01am
Pfizer

Pfizer punts pair of orphan drugs amid rare disease R&D rethink

Jan 31, 2023 9:15am
boardroom

Stealth granted FDA meeting to revive elamipretide filing

Jun 14, 2022 10:10am
Gene therapy

Selecta clinical hold on gene therapy lifted

Mar 10, 2022 10:44am

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