Therapeutic Strategies for Spinocerebellar Ataxia Type 1 (SCA1)
#Neuroscience #Brain #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Cerebellum #Ataxia #SpinocerebellarAtaxia #SCA1 #HereditaryAtaxias #Ataxias #NeurogeneticDiseases #Neurogenetics
#Genetics #HereditaryDiseases #GeneticDiseases
#Biomedicine #Health
#health #biomedicine #GeneticDiseases #HereditaryDiseases #genetics #neurogenetics #NeurogeneticDiseases #Ataxias #HereditaryAtaxias #SCA1 #SpinocerebellarAtaxia #ataxia #Cerebellum #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #brain #neuroscience
Multifaceted nanoparticles: emerging mechanisms and therapies in neurodegenerative diseases.
Friedreich’s ataxia represents a test case to validate the potential of therapeutic strategies based on nanoparticles.
#Neuroscience #Brain #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Cerebellum #Ataxia #FriedreichAtaxia #HereditaryAtaxias #Ataxias #NeurogeneticDiseases #Neurogenetics
#Genetics #HereditaryDiseases #GeneticDiseases
#ClinicalTrial
#GeneTherapy
#Neuroinflammation
#Mitochondria #MitochondrialDysfunction
#Nanoparticles #Nanomedicine #Nanobiotechnology
#Biomedicine #Health
https://academic.oup.com/brain/article/doi/10.1093/brain/awad014/7024724
#health #biomedicine #Nanobiotechnology #Nanomedicine #nanoparticles #MitochondrialDysfunction #mitochondria #neuroinflammation #genetherapy #ClinicalTrial #GeneticDiseases #HereditaryDiseases #genetics #neurogenetics #NeurogeneticDiseases #Ataxias #HereditaryAtaxias #FriedreichAtaxia #ataxia #Cerebellum #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #brain #neuroscience
Vatiquinone found to slow Friedreich's Ataxia progression by 75% after 1.5 years
But oral therapy fails to meet primary goal in Phase 2/3 trial
#Neuroscience #Brain #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Cerebellum #Ataxia #FriedreichAtaxia #HereditaryAtaxias #Ataxias #NeurogeneticDiseases #Neurogenetics
#Genetics #HereditaryDiseases #GeneticDiseases
#ClinicalTrial
#Vatiquinone #Neuroinflammation
#Biomedicine #Health
https://friedreichsataxianews.com/news/vatiquinone-slows-fa-progression-after-18-months-trial-data/
#health #biomedicine #neuroinflammation #Vatiquinone #ClinicalTrial #GeneticDiseases #HereditaryDiseases #genetics #neurogenetics #NeurogeneticDiseases #Ataxias #HereditaryAtaxias #FriedreichAtaxia #ataxia #Cerebellum #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #brain #neuroscience
New Friedreich's Ataxia mouse model may aid research, therapy development
Mice house largest reported number of GAA repeats in Frataxin gene
#Neuroscience #Brain #Neurology #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Neurogenetics #NeurogeneticDiseases #GeneticDiseases #RareDiseases #Ataxia #FriedreichAtaxia #Frataxin
https://friedreichsataxianews.com/news/new-fa-mouse-model-may-aid-research-therapy-development/
#Frataxin #FriedreichAtaxia #ataxia #RareDiseases #GeneticDiseases #NeurogeneticDiseases #neurogenetics #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #neurology #brain #neuroscience
Spinal Cord Abnormalities Correlate With Disease Severity in FA
Results show utility of MRI as biomarker in tracking disease progression
Damage to the spinal dorsal column seems developmental whereas damage of the corticospinal tract may be both developmental and degenerative in origin.
#Neuroscience #Neurology #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Neurodevelopment #NeurodevelopmentalDisorders #Ataxia #FriedreichAtaxia #SpinalCord #Neurogenetics #NeurogeneticDiseases #GeneticDiseases #RareDiseases
#MagneticResonanceImaging #MRI #Biomarker #BrainImaging
https://friedreichsataxianews.com/news/fa-spinal-cord-abnormalities-correlate-with-disease-severity/
#BrainImaging #Biomarker #mri #MagneticResonanceImaging #RareDiseases #GeneticDiseases #NeurogeneticDiseases #neurogenetics #SpinalCord #FriedreichAtaxia #ataxia #neurodevelopmentaldisorders #neurodevelopment #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #neurology #neuroscience
Replay targets genetic brain disorders with new gene therapy company, Kaleibe, utilising big DNA HSV technology
#Biomedicine #GeneTherapy #GeneDelivery #MolecularTherapy #ViralVectors #HerpesviralVectors #HSV
#Neurogenetics #Parkinson #FriedreichAtaxia #Ataxia #NeurogeneticDiseases #GeneticDiseases #NeurologicalDiseases #NeurodegenerativeDiseases #BrainDiseases #Neurology #Neuroscience
https://replay.bio/news/replay-targets-genetic-brain-disorders
#neuroscience #neurology #BrainDiseases #NeurodegenerativeDiseases #NeurologicalDiseases #GeneticDiseases #NeurogeneticDiseases #ataxia #FriedreichAtaxia #parkinson #neurogenetics #hsv #HerpesviralVectors #ViralVectors #MolecularTherapy #GeneDelivery #genetherapy #biomedicine
Deep Intronic FGF14 GAA Repeat Expansion identified as the most common cause of Late-Onset Cerebellar Ataxia
#Neuroscience #Neurology #Neurogenetics #NeurologicalDiseases #NeurodegenerativeDiseases #RareDiseases #GeneticDiseases #Genetics #Cerebellum #Ataxia
#ataxia #Cerebellum #genetics #GeneticDiseases #RareDiseases #NeurodegenerativeDiseases #NeurologicalDiseases #neurogenetics #neurology #neuroscience
Efficacy of Omaveloxolone in Friedreich's Ataxia: Delayed-Start Analysis of the MOXIe Extension
#Neuroscience #Neurology #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Brain #Cerebellum #RareDiseases #GeneticDiseases #FriedreichAtaxia #Ataxia
https://movementdisorders.onlinelibrary.wiley.com/doi/10.1002/mds.29286
#ataxia #FriedreichAtaxia #GeneticDiseases #RareDiseases #Cerebellum #brain #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #neurology #neuroscience
Neurodegenerative Cerebellar Ataxia
#Neuroscience #Neurology #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Ataxia #RareDiseases #GeneticDiseases
https://journals.lww.com/continuum/fulltext/2022/10000/neurodegenerative_cerebellar_ataxia.12.aspx
#GeneticDiseases #RareDiseases #ataxia #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #neurology #neuroscience
#Neuroscience #Neurology #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Ataxia #RareDiseases #GeneticDiseases
https://journals.lww.com/continuum/fulltext/2022/10000/neurodegenerative_cerebellar_ataxia.12.aspx
#GeneticDiseases #RareDiseases #ataxia #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #neurology #neuroscience
Mitochondrial Ataxias: Molecular Classification and Clinical Heterogeneity
#Neuroscience #Neurology #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #Ataxia #Mitochondria #MitochondrialDiseases #RareDiseases #GeneticDiseases
#GeneticDiseases #RareDiseases #MitochondrialDiseases #mitochondria #ataxia #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #neurology #neuroscience
Spinal cord magnetic resonance imaging and spectroscopy detect early-stage alterations and disease progression in Friedreich ataxia
#Neuroscience #Neurology #NeurologicalDiseases #NeurodegenerativeDiseases #Neurodegeneration #FriedreichAtaxia #Ataxia #SpinalCord #RareDiseases #GeneticDiseases
#GeneticDiseases #RareDiseases #SpinalCord #ataxia #FriedreichAtaxia #Neurodegeneration #NeurodegenerativeDiseases #NeurologicalDiseases #neurology #neuroscience
RNA-Targeting Strategy to Repair Genetic Cause of ALS and Frontotemporal Dementia
A novel compound eliminates disease-causing segments of RNA associated with ALS and dementia, and restores the health of neurons in mouse models
#Neuroscience #Neurology #NeurodegenerativeDiseases #ALS #FTD #Dementia
#Genetics #GeneticDiseases #RareDiseases
#MolecularTherapy #ToxicRNA #RNAtargeting
#Biomedicine
#neuroscience #neurology #NeurodegenerativeDiseases #ALS #FTD #dementia #genetics #GeneticDiseases #RareDiseases #MolecularTherapy #ToxicRNA #RNAtargeting #biomedicine
Low Frataxin, Mitochondria Defects Prompt Cell Model Immune Response
Findings show new pathway that might contribute to #FriedreichAtaxia disease mechanisms.
#Neuroscience #Neurology #NeurodegenerativeDiseases #Ataxia #Mitochondria #Frataxin #ImmuneResponse #Interferon #cardiomyocyte #cardiomyopathy #HeartDisease
#GeneticDiseases #RareDiseases #Genetics
https://friedreichsataxianews.com/news/low-frataxin-mitochondria-defects-prompt-fa-immune-response/
#FriedreichAtaxia #neuroscience #neurology #NeurodegenerativeDiseases #ataxia #mitochondria #Frataxin #ImmuneResponse #Interferon #cardiomyocyte #cardiomyopathy #heartdisease #GeneticDiseases #RareDiseases #genetics