Persistent Effect of Arimoclomol in Patients with Niemann-Pick Disease Type C: 24-Month Results from an Open-Label Extension of a Pivotal Phase 2/3 Study

Authors :: Eugen Mengel
Anna Tylki-Szymańska
Marie Aavang Geist
Esther M. Maier
Bénédicte Héron
Thomas Kirkegaard
Matthias Gautschi
Anne Katrine Andreasen
Simon Day
Stephanie Grunewald
Nikolaj H.T. Petersen
Mireia del Toro
Christine í Dali
Federica Deodato
Thomas Hansen
Marc C. Patterson
Saikat Santra
Paul Harmatz
Thomas Blaettler
Rosalia Maria Da Riol
Sabine Grønborg
Linda Ingemann
Agathe Roubertie
Source :: Web of Science
Publication Year :: 2021
Publisher :: Georg Thieme Verlag KG, 2021.

Subjects :: chemistry.chemical_compound
medicine.medical_specialty
Niemann–Pick disease, type C
chemistry
business.industry
Internal medicine
medicine
In patient
Arimoclomol
Open label
medicine.disease
business
Gastroenterology

ISSN :: 14391899
Database :: OpenAIRE
Journal :: Abstracts of the 46th Annual Meeting of the Society for Neuropediatrics
Accession number :: edsair.doi.dedup.....4daf183de73e9a1093f03ebfedd25d28
Full Text :: https://doi.org/10.1055/s-0041-1739640

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