Design and Outcome Measures of BRAVE, a Phase 3 Study of Omaveloxolone in Pediatric Patients With Friedreich Ataxia
Objective: This phase 3 randomized controlled trial will assess the efficacy and safety of omaveloxolone in children aged 2 to <16 years. Background: Omaveloxolone is…Management of Grisel’s Syndrome with Botulinum Toxin: A Case Report
Objective: To present a case of Grisel’s syndrome (GS), highlighting challenges and the efficacy of botulinum toxin therapy in treatment. Background: Grisel’s syndrome (GS) is…Concomitant Progressive Supranuclear Palsy and Amyotrophic Lateral Sclerosis Versus Motoneuron Phenotype of PSP in Ukrainian Patient
Objective: To provide a description of a rare case of mixed progressive supranuclear palsy (PSP) and amyotrophic lateral sclerosis (ALS) in Ukrainian patient. Background: The…Neuroimaging abnormalities of the striatopallidal regions in people with diabetes: A single-center retrospective study
Objective: To describe the imaging abnormalities affecting striatopallidal regions (SP; caudate, putamen, and globus pallidum) in people with diabetes mellitus (DM). Background: DM can lead…POLG2 Variant Reclassification Reveals Diagnosis in Adult with MSA-C Phenotype
Objective: (1) To emphasize the critical role of genetic evaluation in adult-onset movement disorders (2) to demonstrate that POLG2-related disorders should be considered in the…Atypical Parkinsonian Syndromes Presenting with Homonymous Hemianopsia
Objective: We present here two cases of atypical parkinsonism with visual field loss to help increase recognition and drive research efforts forward to elucidate the…Clinical Deficits, Quality of Life and Caregiver Burden across PSP Phenotypes
Objective: To assess clinical deficits, quality of life and caregiver burden across progressive supranuclear palsy (PSP) phenotypes considering also other variables such as age, sex…Spatial Metabolic Covariance Networks in PSP: Implications for Symptomatology and their Neural Basis
Objective: This study aimed to apply spatial ICA to 18F-fluorodeoxyglucose (FDG) positron emission tomography (PET) data from PSP patients and healthy controls (HCs) to identify…Oligodendrocyte-specific mRNA therapy with lipid nanoparticles
Objective: To develop lipid nanoparticles with high efficiency and specificity to introduce mRNA into oligodendrocytes. To denmonstrate the potential therapy of Krabbe disease as one…The effectiveness and safety of autologous adipose derived stromal vascular components (SVF) in the treatment of multiple system atrophy
Objective: This study aimed to explore the efficacy and safety of autologous adipose - derived stromal vascular fraction (SVF) in treating MSA. Background: Multisystem atrophy…
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