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Friedreich's ataxia

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchStrongest evidence only (44)Meta-analysis (8)Treatment guideline (2)Randomized trial (20)Systematic review (9)Phase 3 study (5)Clinical trial (125)Review article (205)Early study (phase 1 or 2) (14)Case report (57)Laboratory or animal research (47)Commentary or editorial (36)Retracted publication (1) Only studies currently seeking participants (25)

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Publications and studies (1112)

• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Multimodal Analysis of the Visual Pathways in Friedreich's Ataxia Reveals Novel Biomarkers. (2023/06/01) ♡
• Blood RNA transcripts reveal similar and differential alterations in fundamental cellular processes in Alzheimer's disease and other neurodegenerative diseases. (2023/06/01) ♡
• Omaveloxolone (Skyclarys(TM)) for patients with Friedreich's ataxia. (2023/06/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Omaveloxolone: First Approval. (2023/06/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Preparing for Disease-Modification Trials in Degenerative Cerebellar Ataxias: Which Endpoints to Choose? (2023/06/01) ♡
• Sensory ataxia and cardiac hypertrophy caused by neurovascular oxidative stress in chemogenetic transgenic mouse lines. (2023/05/29) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Upside-Down Preference in the Forskolin-Induced In Vitro Differentiation of 50B11 Sensory Neurons: A Morphological Investigation by Label-Free Non-Linear Microscopy. (2023/05/06) ♡
• Differences in spontaneous speech fluency between Parkinson's disease and spinocerebellar ataxia type 3. (2023/05/05) ♡
• Acute frataxin knockdown in induced pluripotent stem cell-derived cardiomyocytes activates a type I interferon response. (2023/05/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Apparent Opportunities and Hidden Pitfalls: The Conflicting Results of Restoring NRF2-Regulated Redox Metabolism in Friedreich's Ataxia Pre-Clinical Models and Clinical Trials. (2023/04/27) ♡
• Mitochondrial Calcium Homeostasis in the Pathology and Therapeutic Application in Friedreich's Ataxia. (2023/04/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Phenotype and management of neurologic intronic repeat disorders (NIRDs). (2023/03/01) ♡
• Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Double blind trial of a deuterated form of linoleic acid (RT001) in Friedreich ataxia. (2023/03/01) ♡
• Clinical Drug-Drug Interaction Between Vatiquinone, a 15-Lipoxygenase Inhibitor, and Rosuvastatin, a Breast Cancer Resistance Protein Substrate. (2023/03/01) ♡
• Patient-Reported Impact of Symptoms in Friedreich Ataxia. (2023/02/21) ♡
• Remember Friedreich ataxia even in a toddler with apparently isolated dilated (not hypertrophic!) cardiomyopathy. Revisited. (2023/02/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. SARS-CoV-2 in patients with Friedreich ataxia. (2023/02/01) ♡
• Selection of synthetic proteins to modulate the human frataxin function. (2023/02/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Evaluation of Cerebellar Ataxic Patients. (2023/02/01) ♡
• Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Efficacy of Omaveloxolone in Friedreich's Ataxia: Delayed-Start Analysis of the MOXIe Extension. (2023/02/01) ♡
• Clinical, neuroimaging and genetic findings in children with hereditary ataxia: single center study. (2023/02/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. A new FRDA mouse model [Fxn (null):YG8s(GAA) > 800] with more than 800 GAA repeats. (2023/01/26) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Brain-protective mechanisms of autophagy associated circRNAs: Kick starting self-cleaning mode in brain cells via circRNAs as a potential therapeutic approach for neurodegenerative diseases. (2023/01/16) ♡
• Deep Intronic FGF14 GAA Repeat Expansion in Late-Onset Cerebellar Ataxia. (2023/01/12) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. A promising mouse model for Friedreich Ataxia progressing like human patients. (2023/01/05) ♡
• An intronic GAA repeat expansion in FGF14 causes the autosomal-dominant adult-onset ataxia SCA50/ATX-FGF14. (2023/01/05) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. The inherited cerebellar ataxias: an update. (2023/01/01) ♡
• Progressive Spinal Cord Degeneration in Friedreich's Ataxia: Results from ENIGMA-Ataxia. (2023/01/01) ♡
• Frataxin deficiency alters gene expression in Friedreich ataxia derived IPSC-neurons and cardiomyocytes. (2023/01/01) ♡
• Multiway sparse distance weighted discrimination. (2023/01/01) ♡
• Disease Delineation for Multiple Sclerosis, Friedreich Ataxia, and Healthy Controls Using Supervised Machine Learning on Speech Acoustics. (2023/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients (2023-10-23) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension (2023-09-22) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Coronary Artery Disease in Patients With Friedreich's Ataxia (2023-09-08) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. NAD+ Precursor Supplementation in Friedreich's Ataxia (2023-07-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients (2023-07-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (2023-07-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia (2023-03-28) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. TCA Cycle in the Dentate in Friedreich's Ataxia (2023-03-27) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Early and Longitudinal Assessment of Neurodegeneration in the Brain and Spinal Cord in Friedreich's Ataxia (2023-03-09) ♡
• Clinical, paraclinical and genetic aspects of autosomal recessive cerebellar ataxias (ARCA) in Mali. (2022/12/26) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. S-Glutathionylation and S-Nitrosylation in Mitochondria: Focus on Homeostasis and Neurodegenerative Diseases. (2022/12/13) ♡
• The mutational dynamics of short tandem repeats in large, multigenerational families. (2022/12/12) ♡
• Retinal and Visual Pathways Involvement in Carriers of Friedreich's Ataxia. (2022/12/12) ♡
• G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects. (2022/12/09) ♡
• Ataxia Rating Scales: Content Analysis by Linking to the International Classification of Functioning, Disability and Health. (2022/12/05) ♡
• The Responsiveness of Gait and Balance Outcomes to Disease Progression in Friedreich Ataxia. (2022/12/01) ♡
• Unravelling the etiology of sporadic late-onset cerebellar ataxia in a cohort of 205 patients: a prospective study. (2022/12/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Comorbidities in Friedreich ataxia: incidence and manifestations from early to advanced disease stages. (2022/12/01) ♡
• Harmonizing results of ataxia rating scales: mFARS, SARA, and ICARS. (2022/12/01) ♡

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