Design and Outcome Measures of BRAVE, a Phase 3 Study of Omaveloxolone in Pediatric Patients With Friedreich Ataxia
Objective: This phase 3 randomized controlled trial will assess the efficacy and safety of omaveloxolone in children aged 2 to <16 years. Background: Omaveloxolone is…Feasibility and Preliminary Efficacy of a Pilot Telehealth Physical Activity Intervention in Degenerative Cerebellar Disease (Engage-Ataxia)
Objective: This study evaluated the feasibility and acceptability of the Engage intervention in people with degenerative cerebellar disorders (DCD). Background: Physical activity (PA) can be…Safety and Efficacy of Vatiquinone Treatment in Friedreich Ataxia Patients from MOVE-FA: a Phase 3, Double-blind, Placebo-controlled Trial
Objective: Describe the results of vatiquinone from MOVE-FA (NCT04577352), the 72-week placebo-controlled phase 3 study of patients with Friedreich Ataxia (FA). Background: FA, the most…Improvement in Upright Stability subscale of mFARS with Vatiquinone Treatment in MOVE-FA: a Phase 3, Double-blind, Placebo-controlled Trial
Objective: Describe the results of vatiquinone on the Upright Stability Subscale (USS) from MOVE-FA (NCT04577352), the 72-week placebo-controlled phase 3 study of patients with Friedreich…Deep Brain Stimulation of bilateral ventral intermediate nucleus in a patient with spinocerebellar ataxia type 12
Objective: To treat the medication-resistant tremor of SCA12 by Deep brain stimulation (DBS) Background: Action tremor might be the most prominent feature of SCA12 patient…4-Aminopyridine improves downbeat nystagmus in SCA27b: A case report
Objective: To assess objective clinical improvement in video-oculography in a patient with SCA27b on 4-aminopyridine (4-AP) treatment. Background: Spinocerebellar ataxia 27b (SCA27b; MIM 620174) is…Dentate Deep Brain Stimulation for Cerebellar Ataxia: Long Term Outcomes
Objective: To evaluate long-term results of Deep Brain Stimulation (DBS) targeting the dentate nucleus (DN) in patients with cerebellar ataxia Background: Cerebellar ataxia remains a…Ataxia and Gait Improvement in Relapsing-Remitting Multiple Sclerosis After Transcranial Magnetic Stimulation (TMS): A Case Study
Objective: To evaluate the effectiveness of Transcranial Magnetic Stimulation (TMS) in improving ataxia and gait in a patient with Relapsing-Remitting Multiple Sclerosis (RRMS). Background: Multiple…Evaluating Pharmacological Interventions for Friedreich Ataxia: A Network Meta-Analysis of Randomised Trials
Objective: To compare the clinical efficacy of different pharmacological agents in reduction of disease severity of Friedreich Ataxia. Background: Friedreich ataxia is a rare, inherited,…Predicting Disease Severity in Friedreich’s Ataxia through Wearable Monitoring and Machine Learning.
Objective: Develop and validate a wearable-based solution for tracking motor function in Friedreich ataxia (FRDA). Background: FRDA is an autosomal recessive neurodegenerative disorder causing progressive…
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