Reflection on clinical and methodological issues in rare disease clinical trials.

Johan Verbeeck(Hasselt University), Georg Zimmermann(University of Salzburg), Andrew C. Hooker(Uppsala University), Martin Laimer(Paracelsus Medical University), Geert Molenberghs(University of Antwerp), Joakim Nyberg(Uppsala University), Konstantin Emil Thiel(Paracelsus Medical University), Johann Bauer(Universität Innsbruck), Martin Geroldinger(Paracelsus Medical University), Arne C. Bathke(University of Salzburg)
Orphanet Journal of Rare Diseases
June 5, 2025
10.1186/s13023-025-03805-1
Cited by 1

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