Doc. MUDr. Hana Ošlejšková, Ph.D.
An Open-Label Extenzion of Study HGT-MLD-070 Evaluating Long-Trem Safety of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrofy
č. studie: HGT-MLD-071
MUDr. Pavlína Danhofer, Ph.D.
Účinnost a bezpečnost bumetanidu, perorálního roztoku, podávaného dětem ve věku od dvou do méně než sedmi let s poruchami autistického spektra
č. studie: CL3-95008-002
MUDr. Michal Ryzí, Ph.D.
a) Open-label, 2 dose level trial to evaluate pharmacokinetics, safety, and tolerability od eslicarbazepine acetate (ESL) as adjunctive therapy in infants with refractory epilepsy with partial-onset seizures aged from 1 month to <2 years
b) Open-label, 2 dose level trial to evaluate pharmacokinetics, safety, and tolerability od eslicarbazepine acetate (ESL) as adjunctive therapy in infants with refractory epilepsy with partial-onset seizures aged from 1 month to <2 years - 1 year extension
č. studie: a) BIA-2093-211; b) BIA-2093-211/EXT
MUDr. Lenka Juříková
A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045and SRP-4053 in Patients With Duchenne Muscular Dystrophy
č. studie: 4045-301
MUDr. Lenka Juříková
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
č. studie: VBP15-004/VISION_DMD/
MUDr. Lenka Juříková
Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care - CZE121
č. studie: PTC124-GD-025o-DMD