Efficacy and safety of N-acetyl-l-leucine in Niemann–Pick disease type C

OBJECTIVE: To investigate the safety and efficacy of N-acetyl-l-leucine (NALL) on symptoms, functioning, and quality of life in pediatric (≥ 6 years) and adult Niemann–Pick disease type C (NPC) patients. METHODS: In this multi-national, open-label, rater-blinded Phase II study, patients were assesse...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2021
Materias:
Original Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8361244/
https://www.ncbi.nlm.nih.gov/pubmed/34387740
http://dx.doi.org/10.1007/s00415-021-10717-0

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