The FOsmetpantotenate Replacement Therapy (FORT) randomized, double-blind, Placebo-controlled pivotal trial: Study design and development methodology of a novel primary efficacy outcome in patients with pantothenate kinase-associated neurodegeneration

BACKGROUND/AIMS: Pantothenate kinase-associated neurodegeneration is a rare neurodegenerative disease with a variable clinical phenotype. Fosmetpantotenate is in clinical development as a replacement therapy that targets the underlying cause of pantothenate kinase-associated neurodegeneration. The F...

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Dettagli Bibliografici
Pubblicato in:Clin Trials
Autori principali: Klopstock, Thomas, Escolar, Maria L, Marshall, Randall D, Perez-Dueñas, Belen, Tuller, Sarah, Videnovic, Aleksandar, Greblikas, Feriandas
Natura: Artigo
Lingua:Inglês
Pubblicazione: SAGE Publications 2019
Soggetti:
Design
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6661719/
https://ncbi.nlm.nih.gov/pubmed/31055958
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1740774519845673
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