• A new UK-based research project to understand ‘normal breathing’ in under 6’s calls for participants to help our XLMTM community
    About the “Breathing Muscle Strength in Healthy Children” Study at Brunel University London Brunel University London is involved in an […]
  • The First Cork Hope Walk
    It really didn’t matter that it rained on and off, nothing could spoil the good mood of the walkers who had come along for the first Cork Hope Walk. The 8km walk, which was arranged by Orla Lynch and Stephen Stack, took us along the beautiful Rochestown to Blackrock coastal path. The views were stunning despite the grey sky, and we even got to admire Blackrock Castle where we stopped for the obligatory group photos. […]
  • Fundraising For Ruben
    Over 230 guests attended a hugely successful evening event that was arranged by Siân Carson whose gorgeous one year old son, Rubén, has myotubular myopathy. For the price of their tickets guests got to enjoy a beautifully decorated venue, lovely food, great music and endless great opportunities to raise funds. And boy did they do an amazing job of raising funds… […]
  • The Devon Hope Walk turns into a series of walks, raising over £1300 for Myotubular Trust!
    This year we tentatively planned a fourth Devon Hope Walk for the Myotubular Trust for early September, hoping everyone could make it, after once […]
  • Pretty Muddy Race For Life 2019
    Massive thanks and congratulations to Sarah, Emily and Tara, who recently took part in the 10K Pretty Muddy Race for Life at Sefton Park, Liverpool. […]

What We Do

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.

Family Support

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.


Where it all began, 2006. (Left to right: Professor Victor Dubowitz, Professor Francesco Muntoni, Wendy Hughes and Anne Lennox).

Just some of our achievements:

Between 2006 and 2018

In 13+ years we raised in excess of £2M and funded 14 major research grants internationally.


We held a fifth Conference in 2018 – bringing together patients and their families and carers, researchers and medical professionals to exchange ideas and support.


After a period of intense due diligence we established a Scientific Advisory Board and set up a world class peer review process. Read about our peer review here.

We put out our first Call for Grants.


We were the first disease specific charity to fund gene therapy proof of principle research, which is now the first approved clinical trial for x-linked myotubular myopathy. Dr Ana Buj-Bello, Genethon, Paris.

We funded the very first of our centronuclear myopathy (CNM) research projects.


We funded high throughput gene sequencing for centronuclear myopathies, which ultimately contributed to the discovery of two new genes causing the condition.  These new genes help families with their family planning decisions, and has highlighted researchers to other avenues of investigation. Dr Johann Bohm, IGBMC, France.


We partnered with Sparks, the children’s medical research charity.


We developed and launched the first ever disease specific self-reporting patient registry, The Myotubular and Centronuclear Myopathy Patient Registry.


Funding began on research to develop a drug treatment to reduce levels of the phosphoinositide PI(3)P. Dr James Dowling, Toronto Hospital For Sick Kids, Canada. 

We provided sponsorship for a TREAT-NMD Advisory Committee for Therapeutics (TACT) meeting, where a potential treatment for x-linked myotubular myopathy was evaluated by independent neuromuscular academic reviewers.


We funded a grant to develop the first ever “cross therapy” approach for any myopathy. Dr Jocelyn Laporte, IGBMC, France.

We partnered with TREAT-NMD to host The Myotubular and Centronuclear Myopathy Patient Registry at the University of Newcastle.


We held our first joint European Family Conference with our friends in Germany ZNM-Zusammen Stark!

With sponsorship from Tradeweb, we made our 10th anniversary film.


We partnered with Great Ormond Street Hospital Children’s Charity for grant management.

We were invited to join the Patient Group Consultative Forum of the MHRA UK (Medicine and Healthcare Products Regulatory Agency).

We announced a joint grant call with the renowned children’s research charity Action Medical Research for a £200,000 shared grant in 2018.

A BBC Radio 4 Charity Appeal in support of the Trust was presented by stage and screen actor, Nicholas Farrell.


In January, we sponsored a Standards of Care meeting about centronuclear myopathies for clinicians, patients and their healthcare professionals at the world-renowned Royal Brompton Hospital, London. We sponsored open access for a new publication “Airways clearance techniques in neuromuscular disorders : A state of the art review”

We held a fifth Family Conference in July, jointly with our friends at ZNM-Zusammen Stark!

Since founding, every penny raised by our supporters has been ring fenced to pay for research.  We have been able to raise the money for our running costs separately, with the kind support of specific corporate donations and grants from other Trusts and Foundations.

In June 2010 the Trust became incorporated as a company limited by guarantee (07260229) registered in England and Wales.

Myotubular Trust recognises that the duty of care to safeguard the welfare of children and vulnerable adults is very important. Please see our Safeguarding Policy here.


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