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Anzahl der Publikationen: 3
Zeitschriftenartikel
Klopstock, Thomas; Escolar, Maria L.; Marshall, Randall D.; Perez-Duenas, Belen; Tuller, Sarah; Videnovic, Aleksandar und Greblikas, Feriandas
(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.
In: Clinical Trials, Bd. 16, Nr. 4: S. 410-418
[PDF, 263kB]
Marshall, Randall D.; Collins, Abigail; Escolar, Maria L.; Jinnah, H. A.; Klopstock, Thomas; Kruer, Michael C.; Videnovic, Aleksandar; Robichaux-Viehoever, Amy; Burns, Colleen; Swett, Laura L.; Revicki, Dennis A.; Bender, Randall H. und Lenderking, William R.
(2019):
Diagnostic and clinical experience of patients with pantothenate kinase-associated neurodegeneration.
In: Orphanet Journal of Rare Diseases, Bd. 14, 174
[PDF, 891kB]
Marshall, Randall D.; Collins, Abigail; Escolar, Maria L.; Jinnah, H. A.; Klopstock, Thomas; Kruer, Michael C.; Videnovic, Aleksandar; Robichaux-Viehoever, Amy; Swett, Laura; Revicki, Dennis A.; Bender, Randall H. und Lenderking, William R.
(2019):
A Scale to Assess Activities of Daily Living in Pantothenate Kinase-Associated Neurodegeneration.
In: Movement Disorders Clinical Practice, Bd. 6, Nr. 2: S. 139-149