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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
Bremova-Ertl, Tatiana; Claassen, Jens; Foltan, Tomas; Gascon-Bayarri, Jordi; Gissen, Paul; Hahn, Andreas; Hassan, Anhar; Hennig, Anita; Jones, Simon A; Kolnikova, Miriam; Martakis, Kyriakos; Raethjen, Jan; Ramaswami, Uma; Sharma, Reena; Schneider, Susanne A (2022). Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C. Journal of neurology, 269(3), pp. 1651-1662. Springer 10.1007/s00415-021-10717-0
