Rebecca Storey and Shazia Ahmed, our senior and junior trials coordinators respectively, have been busy writing all the regulatory documents needed for the trial to go ahead. These were submitted to the Health Regulatory Authority in early January. The ethics committee met in early February. This was in Glasgow, but we were allowed to join […]
January 2018 One of the global health priorities of the International Rare Diseases Research Consortium (IRDiRC, www.irdirc.com) is on to find new treatments for the 80% of rare diseases that currently have no cure or means to treat. We know of over 8,000 rare diseases, so this is a really big challenge. As everyone knows, […]
Three British and one Australian research teams, coordinated by Professor Timothy Barrett, have been awarded the funding of 200,000 € by the Association syndrome de Wolfram, the Eye Hope Foundation and the Snow Foundation. Their project aims at “developing biomarkers that will show early evidence of benefit of a treatment; and patient self-report outcome measures […]
Dear Friends and colleagues, I would like to give you an update as to progress with our clinical trial of a treatment for Wolfram syndrome. We had the final confirmation of funding from the UK Medical Research Council on December 16th 2016, so were able to start work on the study protocol early in the […]
Dear Friends and colleagues, I have been fortunate to visit the French Wolfram Association meeting in April in Paris; and the Spanish Wolfram association meeting in May in Almeria. I was humbled by the friendship and community spirit of these families; and they were very tolerant of my poor French and Spanish language skills. On […]
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