Therapies, Research Funding and Racial Diversity in Cerebellar Ataxia: A Systematic Review of the Literature
Objective: To understand the scope of clinical research in cerebellar ataxia by performing a systematic review of controlled clinical trials over the past 50 years,…Isolated ZIC4 antibody associated paraneoplastic cerebellar degeneration
Objective: Anti-ZIC4 antibodies have recently been associated with paraneoplastic cerebellar degeneration (PCD) and are usually associated with small cell carcinoma lung. However, most of the…Gene Expression Quantification to Assess Frataxin Replacement Therapies in Friedreich’s Ataxia
Objective: To identify genetic disease and efficacy markers for Friedreich’s ataxia (FRDA) Background: FRDA is a genetic disease caused by decreased expression of the mitochondrial…Leading Therapeutic Molecules Target Transcription Factors Binding to Repressor Region in FXN Gene
Objective: To investigate interactions of transcription factors (TFs) binding to repressor region located upstream to FXN gene with leading therapeutic molecules. Background: Low levels of…Tissue Frataxin Increases After Administration of CTI-1601, a Frataxin Replacement Therapy in Development for the Treatment of Friedreich’s Ataxia
Objective: To evaluate the pharmacodynamic (PD) and safety profiles of subcutaneous (SC) administration of multiple ascending doses of CTI-1601 in patients age ≥18 years with…Cerebellar bioenergetic depletion following ubiquinol supplementation in a patient with COQ8A-related ataxia
Objective: To non-invasively map the individual treatment response in a patient with COQ8A-related ataxia following coenzyme Q10 supplementation. Background: Primary coenzyme Q10 (CoQ10) deficiency is…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:…Effects of deep transcranial magnetic stimulation of the cerebellum on cerebellar ataxias: A randomized, double-blind, cross-over clinical trial
Objective: To investigate whether cerebellar deep repetitive transcranial magnetic stimulation (d-rTMS) can improve ataxic symptoms when compared to sham stimulation. Background: Cerebellar ataxia remains a…A randomized, sham-controlled, crossover trial on Cerebello-Spinal Transcranial pulsed current stimulation (tPCS) in Ataxia
Objective: Patients of degenerative cerebellar ataxia present with slurring of speech, imbalance during walking and problem of co-ordination in limbs. Currently no effective treatment is…
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