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...

This survey is a patient-led initiative conducted by the Canadian Organization for Rare Disorders. You have been invited to answer this questionnaire because of your involvement as an engaged patient or patient advocate. Your answers will help us understand current knowledge about biosimilars (i.e.  a biologic drug demonstrated to be...

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...

Lefil, Université Laval’s journal, published an article presenting the new advances of its researchers to improve the effectiveness of the CRISPR-Cas tool’s in the gene therapy. This research, in collaboration with the Dalhousie University, aims to develop a new strategy to increase the effectiveness of the CRISPR-Cas tool by modifying...

On April 15, the newspaper Le Devoir published three articles on gene therapy to which Dr. Jacques P. Tremblay, researcher of our scientific committee, talks about the progress of this treatment. These three papers describe gene therapy, scientific advances, economic needs and opportunities for Quebec in relation to the development...