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...
The following text comes from this article: https://friedreichsataxianews.com/2017/03/16/study-cells-friedreichs-ataxia/ “The selective degeneration of certain neurons in patients with Friedreich’s ataxia may be explained by the effect that the lack of frataxin has on nervous system cells called astrocytes. Researchers discovered that mice, which grew up without frataxin, had abnormal astrocytes in their cerebellum,...
The team of Dr. Jacques P. Tremblay (Research Center of the CHU of Quebec – Université Laval) is continuing his research to develop a treatment for Friedreich’s Ataxia. Dr. Tremblay’s team recently obtained a grant from Ataxia Canada to develop a new AAV serotype capable of infecting the brain and...
