We want to present the research of Martine Tétreault Assistant professor Department of neuroscience, University of Montreal The identification of the genetic causes and mechanisms associated with rare Mendelian diseases has always been an important challenge for the medical field. The identification of mutations and/or genes associated with a disease...
Current and emerging therapies for Friedrich’s Ataxia (FA) focus on reversing the deleterious effects of such deficiency including mitochondrial augmentation and increasing frataxin levels, providing the possibility of treatment options for this physiologically complex, multisystem disorder. In this review article, the authors discuss the current and prior in vivo and...
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,...
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...
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...
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...
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...