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,…Transitional Life Events in Friedreich Ataxia: Differential Age at Onset Perspectives
Objective: To identify and quantify the age-contingent impact of Friedreich’s Ataxia. Background: Friedreich's ataxia (FA) is a rare, inherited multisystem neurodegenerative disorder that primarily affects…Effects of a Six-Week Neurorehabilitation Program on Ataxia, Balance and Gait in a Patient with CANVAS Syndrome: A Case Report
Objective: This study aimed to examine the effects of a neurologic physiotherapy on ataxia, balance, and gait in a patient with cerebellar ataxia, neuropathy, and…Healthcare Utilization and Economic Burden in Patients with Friedreich’s Ataxia: Baseline Results of the PROFA Study
Objective: The study aimed to investigate healthcare utilization, costs, and the distribution of costs across ambulation levels among patients with Friedreich's ataxia. Background: Friedreich's ataxia…An interesting case of progressive vision loss and unusual gait
Objective: To highlight the importance of vision diminution and improvement with drugs a clue for treatable disorder like abetalipoproteinemia. Background: A 22 years old male…
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