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Bremova-Ertl, Tatiana; Schneider, Susanne (2023). Current advancements in therapy for Niemann-Pick disease: progress and pitfalls. Expert opinion on pharmacotherapy, 24(11), pp. 1229-1247. Informa Healthcare 10.1080/14656566.2023.2215386
Martakis, Kyriakos; Claassen, Jens; Gascon-Bayari, Jordi; Goldschagg, Nicolina; Hahn, Andreas; Hassan, Anhar; Hennig, Anita; Jones, Simon; Kay, Richard; Lau, Heather; Perlman, Susan; Sharma, Reena; Schneider, Susanne; Bremova-Ertl, Tatiana (2023). Efficacy and Safety of N-Acetyl-L-Leucine in Children and Adults With GM2 Gangliosidoses. Neurology, 100(10), e1072-e1083. American Academy of Neurology 10.1212/WNL.0000000000201660