Fast and controlled access to tofersen for SOD1 -related amyotrophic lateral sclerosis in The Netherlands; experiences using the orphan drug access protocol

Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2026

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Authors: Sean W. Willemse, Carla E. M. Hollak, Niels D. Reijnhout, Carelle C. Reparon-Schuijt, Ankoor Roy, Koen C. Demaegd, Ruben P. A. van Eijk, Leonard H. van Den Berg, Michael A. van Es
DOI: 10.1080/21678421.2026.2655738
Cited by: 1
References: 8

Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disorder characterized by muscle weakness and spasticity. Prognosis is poor, with death due to respiratory insuffici...

Fields of Study & Subjects

Amyotrophic Lateral Sclerosis Research Neurogenetic and Muscular Disorders Research Peripheral Neuropathies and Disorders

Publication details

Journal / source
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Publisher
Informa UK Limited
Pages
1–3
Publication date
13 April 2026
Language
EN