A translational approach to determine target concentrations of MIN-102 (leriglitazone) to support a Phase II study in Friedreich’s ataxia
Objective: To determine efficacious plasma concentration of leriglitazone from preclinical models of Friedreich´s ataxia (FRDA). To confirm CNS target engagement in a first-in human study.…Feasibility and Acceptability of Lee Silverman Voice Treatment for patients with hereditary ataxia
Objective: This study aimed to assess the feasibility and acceptability of Lee Silverman Voice Treatment (LSVT) provided via Skype for patients with hereditary ataxia. Background:…Supportive treatments for Ataxia, a Randomized Control Trial
Objective: The objective of this study is to evaluate the effectiveness of supporting treatments that improve awareness and the quality of patient life and counsel…Exergame training in Early Onset Ataxia patients
Objective: In ambulant EOA children, we aimed to study the effect of a medically designed “ice-skating exergame” on balance, posture and muscle force parameters Background:…Phosphodiesterase inhibitors as a treatment for Friedreich’s ataxia
Objective: To evaluate the effect of phosphodiesterase inhibitors, as potential treatment for Friedreich’s ataxia, in a Drosophila melanogaster model of the disease. Background: Friedreich’s ataxia…Normative Reference for Timed Up and Go Test (TUG) in Young Indian Adults- Preliminary Results
Objective: This study investigated the clinical usefulness of the time up and go test in young population and provide a reference range for healthy adults.…Clinical and molecular features of a Chinese family with spinocerebellar ataxia type 6
Objective: To evaluate the clinical manifestation, CAG repeats of abnormal alleles, mitochondrial function and treatment of spinocerebellar ataxia type 6To evaluate the clinical manifestation, CAG…Think of Gluten Ataxia:A rare but potentially reversible cause of progressive neurological disorder
Objective: To explore an unusual cause of ataxia in a patient with known Celiac disease. Background: Celiac disease is the established but not the only…A case of cerebellar ataxia caused by Pembrolizumab for Non-small cell lung cancer
Objective: We reported a case that cerebellar ataxia and decreased accumulation of right cerebellum at SPECT appeared after immunotherapy using immune-checkpoint inhibitor “Pembrolizumab” for Non-small…Inpatient vs outpatient workup of patients with ataxia and suspected paraneoplastic cerebellar degeneration: Does work-up location matter?
Objective: To assess the outcomes of patients with ataxia evaluated for paraneoplastic syndrome primarily in an outpatient or inpatient setting. Background: Although paraneoplastic ataxias are…
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