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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Detalles Bibliográficos
Autores principales: Klopstock, Thomas, Escolar, Maria L, Marshall, Randall D, Perez-Dueñas, Belen, Tuller, Sarah, Videnovic, Aleksandar, Greblikas, Feriandas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2019
Materias:
Design
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6661719/
https://www.ncbi.nlm.nih.gov/pubmed/31055958
http://dx.doi.org/10.1177/1740774519845673

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6661719/
https://www.ncbi.nlm.nih.gov/pubmed/31055958
http://dx.doi.org/10.1177/1740774519845673

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