See the presentations from our Scientific Webinar 27th November

We were honoured to have had keynote speakers join us to present the scientific updates at our Myotubular and Centronuclear Myopathy Webinar event last Saturday. We were delighted too that so many families could join us for this UK event.

Professor Muntoni provided everyone with an update on the Astellas Gene Therapy’s clinical trial ASPIRO, he discussed ‘what we have learned‘ specifically around the liver problems recently seen in gene therapy. Dr Chris Freitag, CMO of Dynacure presented an update on ‘antisense therapy’ in the ‘UNITE-CNM’ adult trial for MTM1 and DYN2 patients, and spoke about the preparations being made to potentially extend it to a children’s trial in the next Phases. Professor Quinlivan provided additional information about the UK sites and the challenges encountered by COVID-19 since 2019, for the UNITE-CNM study patients involved. And finally, Dr Giovanni Baranello provided an update about the repurposing of the drug tamoxifen in the TAM4MTM trials which is for children under the age of 18 years and is planned to begin in the UK site, Great Ormond Street Hospital.

You can find each of the presentations from Saturday by scrolling down (PPX). Please email us with any specific questions you may have, but please be aware that Dynacure regularly update their Frequently Asked Questions section of their website which is available in different languages. https://www.dynacure.com/patients-and-families/

Anne Lennox gave an overview of the regulatory process of bringing an ‘investigational product’ (or drugs being tested) in clinical trial to license – where it would be available for all patients.

Orla Lynch and Wendy Hughes introduced the new Home Care survey which will hopefully provide experiences and insights into the way home care is currently delivered to our UK patient community. The survey is due to be launched in early 2022 and more details will be provided about how to participate once it is open.

We would like to thank everyone who has supported Myotubular Trust this year, and in previous years. Without your support we would not be where we are today, with, currently, three independent trials for MTM/CNM. We hope that, in future, there will be even more trials emerging for new investigational products to treat or ‘cure’ patients living with MTM/CNM, which will be as a direct result of the research you are kindly helping us to support with funding.

Please find out how you can regularly donate here to support this ongoing work. Thank you.