ArticleClinical trials (London, England)2019

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.

Thomas Klopstock et al.PubMed ↗Full text ↗Publisher ↗

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