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1.
Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration.
Mov Disord
; 36(6): 1342-1352, 2021 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-33200489
2.
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.
Clin Trials
; 16(4): 410-418, 2019 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-31055958
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