Gene therapy abroad

Neurocrine Biosciences and Voyager Therapeutic announced the formation of a strategic collaboration focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine Biosciences’ expertise in neuroscience,...

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A new Swedish qualitative study on the impact of familial ataxias on persons’ life (June 2017)

The article Cerebellar ataxia and intrathecal baclofen therapy: Focus on patients’ experiments, published in the online journal PLOS ONE, presents the results of a qualitative research on the impact of family ataxias on persons’ life. “The aim of this study was to elucidate and better understand patients´ experiences of living...

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Friedreich’s Ataxia News present a study from University of Bristol in the United Kingdom about a cytokines therapy that could potentially decrease the symptoms of Friedreich’s ataxia (February 2017)

Resume from the Friedreich’s Ataxia News article, Specific Cytokines Therapy Protected Neurons in Humanized Mouse Study “A cocktail of clinically approved cytokines for other diseases shows promising neuro-protective effects in a Friedreich’s ataxia (FA) humanized mouse model. The study, “Cytokine therapy-mediated neuroprotection in a Friedreich’s ataxia mouse model” was published...

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Friedreich’s Ataxia News present a new study on a vibration device that could help movement coordination for ataxic persons

Resume from the Friedreich’s Ataxia News article, Vibration Device, Equistasi, Seen to Aid Movement in Ataxia Patients in Small Study  “The study, “A Wearable Proprioceptive Stabilizer For Rehabilitation Of Limb And Gait Ataxia In Hereditary Cerebellar Ataxias: A Pilot Open-Labeled Study,” was published in the journal Neurological Sciences. Equistasi is...

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Friedreich’s Ataxia News present a new study on the impacts of cerebellar atrophy on other brain structures

Resume from the Friedreich’s Ataxia News article, Structural Changes May Serve as Biomarker for Ataxia, Study Finds   “Researchers at Italy’s IRCCS Santa Lucia Foundation have found that cerebellar atrophy can affect brain structures related to emotions, thinking and memory, which may in part explain ataxia symptoms. Their results suggest that...

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Liver Growth Factor (LGF) Upregulates Frataxin Protein Expression and Reduces Oxidative Stress in Friedreich’s Ataxia Transgenic Mice

Friedreich’s ataxia (FA) is a severe disorder with autosomal recessive inheritance that is caused by the abnormal expansion of GAA repeat in intron 1 of FRDA gen. This alteration leads to a partial silencing of frataxin transcription, causing a multisystem disorder disease that includes neurological and non-neurological damage. Recent studies...

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