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Events
MDUK Patient Information Days 2025
Muscular Dystrophy UK are hosting free patient information days, providing an opportunity to connect with others affected by muscular dystrophy and learn about vital resources and support available to you. Members of our registries team will be attending the London, Birmingham and Lisburn events to chat about registry participation and support new registrations, so feel free to come and say hello!
Click here to read the message from MDUK about their patient information days including links to registration. Please send any questions or comments directly to MDUK.
ZNM – Zusammen Stark! e.V. Family Conference 2025 - Registration open until 28th February 2025
ZNM – Zusammen Stark! e.V. is celebrating its 10th anniversary and would like to invite patients to join their Family Conference 2025 in Bad Nauheim, Germany, from May 29 to June 1, 2025!
Click here for more information about the event and how to register.
Newsletters
If you would like to receive a copy of the Registry newsletter, please contact us.
- August 2024: Newsletter (opens in new window)
- October 2023: Newsletter (opens in new window)
- September 2022: Newsletter (opens in new window)
- February 2022: Call for Participation in Myotubular and Centronuclear Myopathy Research
- December 2021: Newsletter (opens in new window)
Research News
January 2025 - Astellas Gene Therapies is recruiting for the Epidemiologic Study of XLMTM and Clinical Expression in the Liver (EXCEL) study (NCT06581146)
The registry has contacted participants who may be eligible for the upcoming EXCEL study. The study is an observational, prospective study (participants and certain outcomes will be observed for period of time) using liver-focused assessments. Approximately 50 male participants with XLMTM will be enrolled at specialist sites in the United States, Canada, and the United Kingdom.
Click here to read more about the study inclusion criteria, and how to get involved.
December 2024 - TAM4MTM XLMTM community webinar
On 3rd December 2024, the Myotubular Trust, a UK-based patient organization, hosted a community webinar to share updates on the discontinuation of the TAM4MTM clinical trial. Dr James Dowling, SickKids Hosptial Toronto, is the lead Principal Investigator and Sponsor of the study outlined the objectives and reasons why the study was terminated.
Click here to read more.
November 2024 - TAM4MTM Trial Halted
It is with regret that we share the news of the Tamoxifen trial (TAM4MTM) stopping due to impact on liver function.
Click here to read the letter from Dr Dowling and Dr Baranello, explaining the halting of the trial.
Other News
- June 2023: Newcastle University’s John Walton Muscular Dystrophy Research Centre Contributes Data to C-Path’s RDCA-DAP (opens in new window)