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…Omaveloxolone in a real-world setting: implications in Friedreich Ataxia beyond the classic study population
Objective: The aim was to monitor safety and efficacy of omaveloxolone treatment in a single center real life cohort, focusing on Friedreich Ataxia (FRDA) patients…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…
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