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Eine Ebene höherGruppiert nach: Dokumententyp |
Veröffentlichungsdatum
Anzahl der Publikationen: 3
2021
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 und Schneider, Susanne A.
(2021):
Efficacy and safety of N-acetyl-l-leucine in Niemann-Pick disease type C.
In: Journal of Neurology, Bd. 269, Nr. 3: S. 1651-1662
2020
Donald, Aimee; Tan, Chong Y.; Chakrapani, Anupam; Hughes, Derralyn A.; Sharma, Reena; Cole, Duncan; Bardins, Stanislav; Gorges, Martin; Jones, Simon A. und Schneider, Erich
(2020):
Eye movement biomarkers allow for the definition of phenotypes in Gaucher Disease.
In: Orphanet Journal of Rare Diseases, Bd. 15, Nr. 1, 349
2018
Cortina-Borja, Mario; te Vruchte, Danielle; Mengel, Eugen; Amraoui, Yasmin; Imrie, Jackie; Jones, Simon A.; Dali, Christine I.; Fineran, Paul; Kirkegaard, Thomas; Runz, Heiko; Lachmann, Robin; Bremova-Ertl, Tatiana; Strupp, Michael und Platt, Frances M.
(2018):
Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials.
In: Orphanet Journal of Rare Diseases
13:143
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