The FOsmetpantotenate Replacement Therapy (FORT) randomized, double-blind, Placebo-controlled pivotal trial: Study design and development methodology of a novel primary efficacy ou...

por Klopstock, Thomas, Escolar, Maria L, Marshall, Randall D, Perez-Dueñas, Belen, Tuller, Sarah, Videnovic, Aleksandar, Greblikas, Feriandas
Publicado 2019

Open-Label Fosmetpantotenate, a Phosphopantothenate Replacement Therapy in a Single Patient with Atypical PKAN

por Christou, Yiolanda-Panayiota, Tanteles, George A., Kkolou, Elena, Ormiston, Annita, Konstantopoulos, Kostas, Beconi, Maria, Marshall, Randall D., Plotkin, Horacio, Kleopa, Kleopas A.
Publicado 2017

A Scale to Assess Activities of Daily Living in Pantothenate Kinase‐Associated Neurodegeneration

por 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., Lenderking, William R.
Publicado 2019

Diagnostic and clinical experience of patients with pantothenate kinase-associated neurodegeneration

por 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., Lenderking, William R.
Publicado 2019