Gene Therapy Update for Friedreich’s Ataxia What patients and families should know (March 2026) Who is Lexeo Therapeutics? Lexeo Therapeutics is a biotechnology company developing gene therapies for heart disease, including heart involvement caused by Friedreich’s ataxia (FA)—a major cause of illness and premature death in FA. What treatment is...
The study is being conducted with multiple sites in the United States and Canada seeking to recruit approximately 9 participants aged 18-40 with a diagnosis of FA-associated cardiomyopathy. The study will be conducted by Dr. Antoine Duquette at the CHUM in Montreal. The choice of Montreal as a site reflects...
We are currently considering a new form of funding for Professor Tremblay’s project. Our goal is to develop a mouse model for the research on Friedreich’s Ataxia. The research project will soon be published in the American crowdfundig site CONSANO. Ataxia Canada will contribute equally to the amount collected. Access to...
When armed conflicts, plagues, and cataclysms are making headlines, only few medias cares about the fate of millions of people in the world suffering of hereditary disabilities. Even so, some countries do care about it. The Institute of Genetic Diseases Imagine was inaugurated June 28 2014 in Paris, France. It...
The petition for funding of gene therapy for hereditary diseases in Quebec was proposed by Professor Jacques P. Tremblay, signed by 13 other researchers, and supported by a provincial deputy for Vanier Rivers, Patrick Huot. In the current state of government finances, researchers do not require the addition of new financial...
International Consortium for Gene and Cell Therapy … we have a societal obligation to the rare-disease community to collaborate and build the infrastructure to meet it. With an international consortium in place, it is more likely that therapies will be established that help patients not only in the developed world...