Dr James Dowling of Sick Kids, Toronto, Canada has been a recipient of grants from the Myotubular Trust in both 2014 and 2015 to research a potential treatment for myotubular myopathy. Dr Dowling has been working on proving the effect of reducing levels of the phosphoinositide called PI(3)P on reducing the symptoms of myotubular myopathy. Results to date are very encouraging and this was work published in the high impact journal, JCI The Journal of Clinical Investigations, in August 2016.
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NEWS & UPDATES
- NCB invites you to join an online focus group interview to help improve care for LTV children and young adults in the UKFebruary 13, 2019Do you have approximately 90 minutes to spare to help with an important study that will aim to improve care for long-term ventilated children and […]
- Gene therapy in x-linked myotubular myopathy education resource is launched by the American Society of Gene & Cell TherapyFebruary 12, 2019Patients suffering from x-linked myotubular myopathy (XLMTM) do not have enough of the myotubularin protein, so the body may require a ventilator to […]
- St Joseph’s Primary Academy raise money with their very own ‘Bake Off’February 1, 2019Pupils, staff and parents of St Joseph’s Primary Academy in Cleethorpes, supported a few charities, including Myotubular Trust by holding their very […]
- Audentes Therapeutics announces next steps in the development of their gene therapy for x-linked myotubular myopathyJanuary 31, 2019Today, 31st January, Audentes provided an update on meetings with the FDA (US regulator), planned conversations with the EMA (European regulator) and […]
- Artwork to create new funds for the Myotubular TrustJanuary 24, 2019We are delighted to announce that for a limited period, our long term supporter and established artist, Paul Le-Tallec, is raising funds for […]