We received charitable status in March 2006 and our charitable aims are:
“The relief of disability and the extension of life for those suffering from myotubular myopathy, by promoting the study of, and research into, the treatment and cure of the muscle weakness caused by myotubular myopathy”
Our goal was to become a well known and respected source of funds for international researchers, the existence of which in itself may provide inspiration and focus for innovative science. We wanted to be able to fund proof of principle research projects that would not normally be funded by public or industrial funding. In 2008, after a period of intense due diligence, we set up our medical peer review process and established the Scientific Advisory Board. We made our first call for applications for research projects in September 2008. We have ensured that the review process is nothing short of world class, therefore utilising every penny donated to us in the best possible way. From our very first grant awards to today, we have annually invested in research for centronuclear myopathy as well as the more common x-linked myotubular myopathy. Our aim is to focus on everyone affected by this condition. Ultimately, we wish to find a cure or treatment for myotubular and centronuclear myopathy. The first grant I received from Myotubular Trust was really helping us to start with the project; it was like opening a door. With this money we were able to establish the proof of concept that gene therapy is working in myotubular myopathy – Ana Buj-Bello, Genethon, France. To read more about the very promising research we have funded please read our Research pages.
Having a rare disease, especially one that is life threatening, can be very isolating. Since founding we have supported families by putting them in touch with each other; providing contacts with and advice from leading experts in the condition and helping them with different stages of living with myotubular and centronuclear myopathy. As parents with many years of combined experience in managing the condition, we feel we are in a unique position to provide a real empathy, alongside practical advice. We now feel that at least we are going somewhere with our son’s future plan. Thank you for your support; you are one of the reasons why we are still standing.
History of Myotubular Trust
Anne Lennox and Wendy Hughes, two parents of children affected by myotubular myopathy, set up the Myotubular Trust in February 2006. It was very clear that as a rare condition, research into myotubular myopathy could lag substantially behind the scientific developments in other fields of muscle disease, due to lack of dedicated funds, and the difficulty of “competing” with other more common diseases. The Founding Patron is the renowned Professor Victor Dubowitz, Emeritus Professor of Paediatrics at University of London and President of the World Muscle Society. Professor Francesco Muntoni, Professor of Paediatric Neurology and Head of The Dubowitz Neuromuscular Centre at the Institute of Child Health/Great Ormond Street Hospital for Children, is our scientific advisor and Chair of our Scientific Advisory Board.
In June 2010 the Trust became incorporated as a company limited by guarantee (07260229) registered in England and Wales.