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Eine Ebene höherGruppiert nach: Dokumententyp |
Veröffentlichungsdatum
Anzahl der Publikationen: 5
2021
Thakur, Nivedita; Klopstock, Thomas; Jackowski, Suzanne; Kuscer, Enej; Tricta, Fernando; Videnovic, Aleksandar und Jinnah, Hyder A.
(2021):
Rational Design of Novel Therapies for Pantothenate Kinase-Associated Neurodegeneration.
In: Movement Disorders, Bd. 36, Nr. 9: S. 2005-2016
2020
Klopstock, Thomas 
ORCID: https://orcid.org/0000-0003-2805-4652; Videnovic, Aleksandar; Bischoff, Almut Turid; Bonnet, Cecilia; Cif, Laura; Comella, Cynthia; Correa-Vela, Marta; Escolar, Maria L.; Fraser, Jamie L.; Gonzalez, Victoria; Hermanowicz, Neal; Jech, Robert; Jinnah, Hyder A.; Kmiec, Tomasz; Lang, Anthony; Marti, Maria J.; Mercimek-Andrews, Saadet; Monduy, Migvis; Nimmo, Graeme A. M.; Perez-Duenas, Belen; Pfeiffer, Helle Cecilie Viekilde; Planellas, Lluis; Roze, Emmanuel; Thakur, Nivedita; Tochen, Laura; Vanegas-Arroyave, Nora; Zorzi, Giovanna; Burns, Colleen und Greblikas, Feriandas
(2020):
Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration.
In: Movement Disorders, Bd. 36, Nr. 6: S. 1342-1352
[PDF, 305kB]
ORCID: https://orcid.org/0000-0003-2805-4652; Videnovic, Aleksandar; Bischoff, Almut Turid; Bonnet, Cecilia; Cif, Laura; Comella, Cynthia; Correa-Vela, Marta; Escolar, Maria L.; Fraser, Jamie L.; Gonzalez, Victoria; Hermanowicz, Neal; Jech, Robert; Jinnah, Hyder A.; Kmiec, Tomasz; Lang, Anthony; Marti, Maria J.; Mercimek-Andrews, Saadet; Monduy, Migvis; Nimmo, Graeme A. M.; Perez-Duenas, Belen; Pfeiffer, Helle Cecilie Viekilde; Planellas, Lluis; Roze, Emmanuel; Thakur, Nivedita; Tochen, Laura; Vanegas-Arroyave, Nora; Zorzi, Giovanna; Burns, Colleen und Greblikas, Feriandas
(2020):
Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration.
In: Movement Disorders, Bd. 36, Nr. 6: S. 1342-1352
[PDF, 305kB]
2019
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