Fast and controlled access to tofersen for SOD1-related amyotrophic lateral sclerosis in The Netherlands; experiences using the orphan drug access protocol.
Sean W Willemse, Carla E M Hollak, Niels D Reijnhout, Carelle C Reparon-Schuijt, Anke A M G Pisters-van Roy, Koen C Demaegd, Ruben P A van Eijk, Leonard H van Den Berg, Michael A van Es
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