Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration.
Thomas KlopstockAleksandar VidenovicAlmut Turid BischoffCecilia BonnetLaura CifCynthia ComellaMarta Correa-VelaMaria L EscolarJamie L FraserVictoria GonzalezNeal HermanowiczRobert JechHyder A JinnahTomasz KmiecAnthony LangMaria J MartíSaadet Mercimek-AndrewsMigvis MonduyGraeme A M NimmoBelen Perez-DueñasHelle Cecilie Viekilde PfeifferLluis PlanellasEmmanuel RozeNivedita ThakurLaura TochenNora Vanegas-ArroyaveGiovanna ZorziColleen BurnsFeriandas GreblikasPublished in: Movement disorders : official journal of the Movement Disorder Society (2020)
Treatment with fosmetpantotenate was safe but did not improve function assessed by the PKAN-ADL in patients with PKAN. © 2020 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.