Home-Based Targeted tDCS for Cerebellar Ataxia: Results of a Randomized Double-Blind Crossover Study with Remote Functional Assessment
Objective: To evaluate the clinical efficacy, feasibility, and safety of targeted home-based cerebellar tDCS with remotely supervised functional assessment. Background: Cerebellar ataxia is a disabling…Transcriptomic Profiling of Frataxin-Deficient Drosophila melanogaster to Identify Molecular Drivers and Pathway Disruptions in Friedreich’s Ataxia
Objective: To characterize the global transcriptomic changes and molecular pathways disrupted by frataxin deficiency in a Drosophila model of Friedreich's Ataxia (FA) to identify potential…Synchronous Unilateral Pendular Nystagmus and Palatal Myoclonus After Pontine Hemorrhage: A Case Report
Objective: To present a post-stroke pontine bleed patient who developed a rare movement disorder with a treatment approach. Background: A 43-year-old male presented with involuntary…Pueraria tuberosa improves the locomotion defects in Drosophila model of FRDA
Objective: To study the therapeutic potential of the medicinal herb Pueraria tuberosa on Drosophila model of Friedreich’s ataxia. Background: Reduced levels of the mitochondrial protein…Clinical Profile, Therapeutic Interventions, and Outcome Predictors of Paraneoplastic Movement Disorders – A Tertiary Care Centre Experience
Objective: To analyze the clinical spectrum, associated tumors, antibody profile, outcomes, and prognostic predictors in a cohort of paraneoplastiic movement disorders admitted in a tertiary…Repetitive Transcranial Magnetic Stimulation Improves Static Balance Control in Cerebellar Ataxia
Objective: Repetitive transcranial magnetic stimulation (rTMS) has shown promise in improving motor functions in other movement disorders. This pilot study aimed to investigate the effects…Interventions in Friedreich’s Ataxia and their Influence on Patient-centric Outcomes: A Systematic Review
Objective: The presented systematic review summarizes available pharmacological and non‑pharmacological interventions for patients with Friedreich's ataxia, their outcomes, and alignment with patient‑centered health domains as…Intravenous AAVrh.10hFXN (LX2006) Gene Therapy in Friedreich Ataxia: Early Neurologic Assessment from Two Phase 1 and 2 Studies
Objective: To evaluate neurologic measures of efficacy of intravenous AAVrh.10hFXN (LX2006) gene therapy in adults with Friedreich ataxia, based on data from the SUNRISE-FA study…Positive Results from a Global Phase 3 Trial (IB1001-303) Evaluating Levacetylleucine in Ataxia-Telangiectasia
Objective: To report efficacy and safety findings from the pivotal, Phase 3 trial evaluating levacetylleucine in children and adults with Ataxia-Telangiectasia (A-T). Background: A-T is…A Phase 3, Multinational, Randomized, Placebo-Controlled, Double-Blind, Crossover Trial (IB1001-304) Evaluating Levacetylleucine in CACNA1A Disorders
Objective: To assess the efficacy, safety, and tolerability of levacetylleucine in people with CACNA1A disorders. Background: CACNA1A disorders refer to a range of rare, progressive,…
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