Registries for orphan drugs: generating evidence or marketing tools?

by Carla E. M. Hollak, Sandra Sirrs, Sibren van den Berg, Vincent van der Wel, Mirjam Langeveld, Hanka Dekker, Robin Lachmann, Saco J. de Visser
Published 2020-09-01

A novel sustained‐release cysteamine bitartrate formulation for the treatment of cystinosis: Pharmacokinetics and safety in healthy male volunteers

by Cécile L. Berends, Lisa Pagan, Michiel J. vanEsdonk, Naomi B. Klarenbeek, Kirsten R. Bergmann, Matthijs Moerland, Vincent van derWel, Saco J. deVisser, Hans Büller, Frans deLoos, Wouter S. deVries, Hans Waals, Leo G. J. deLeede, Jacobus Burggraaf, Ingrid M. C. Kamerling
Published 2021-04-01