Research is a big part of what we are involved in. Scientific knowledge is the stepping stone to understanding this disease.

Cure DM facilitate research in many ways:

* We attend conferences, advocating for our community and ensuring the myotonic dystrophy patient and carer is represented.

*  We ensure any and all the information we share is accurate and correct - and up to date!

* We are on multiple panels and collaborate to publish documents and disseminate info.

* We directly support and fund researchers and clinicians.

* We take the patients perspective to those working on studies and researching this complex condition. We remind them that we are real people, and advocate on behalf of the DM community at every opportunity.


It is vital we continue to learn about Myotonic Dystrophy, particularly congenital and early childhood onset, and how it affects our children and loved ones. We aim to make this information common knowledge, and try to find treatments. Congenital DM is the most severe form of DM1 and is very much under represented.

No two people are affected in the same way, no two children present with the same symptoms.

This page will have updates on research.



Cure DM awards grant. June 2022.

Multi-centre audit of Myotonic dystrophy care in the UK: Is it the time for national recommendations?

Litesh., et al

Cure DM are delighted to have recently supported 3rd year medical student Mohit Litesh (working alongside Dr Channa Hewamadduma, Consultant Neurologist in Sheffield) with a grant towards the attendance and presentation of his abstract, for the annual conference for the Association of British Neurologists (ABN).


Click here for more details.

Cure DM research survey



Please click above to add your responses to our survey.

 You can see our ongoing results which have been presented HERE

Cure DM published this questionnaire in 2020, with the aim to gather information from our UK Myotonic Dystrophy community, which can be put together and presented at Myotonic Dystrophy conferences around the world.

We often hear 'you are the only ones in that area' or are told about the services available, however, over the years our charity has come to realise that this is not always the case.

Our ongoing survey aims to support research, day to dare car and inprove services all over the UK,

Please complete the questionnaire and help to show that we are not small in number and we deserve standardised care wherever we live!

(All results are anonomysed and no identifiable information is shared)

Current Trials for Congenital Myotonic Dystrophy.


AMO-02 (Tideglusib) is in trial for the treatment of CDM.

 CLICK HERE to see updates.



Click HERE to learn more about the UK DM Patent registry and to see the current drug/research pipeline.

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PHENODM1 Natural History study

Newcastle and London have now completed the natural history study in Myotonic Dystrophy type 1. (Adults)

Cure DM were delighted to provide funding towards this trial.

This study is now complete, Thank you to all who took part.

This was an observational study and did not involve the trial of any treatment or therapy. This study is important for doctors and researchers to understand as much as possible about Myotonic Dystrophy type 1 and how it affects people in different ways. It will help treatments being developed in the laboratory and being tested in clinical trials in the future,

Visit the CLINICAL TRIALS GOV page, for more detailed information.



Cure DM is delighted to be recognised as an official partner within the Prefer Project consortium.
It is an honour to be recognised for the work we are doing within the NMD sections of the study. Please keep an eye on this page for official updates and information regarding the study.

"PREFER will establish recommendations to support development of guidelines for industry, Regulatory Authorities and HTA bodies on how and when to include patient perspectives on benefits and risks of medicinal products. "  

Click here to read more.


In this project Professor Brook and his team at the University of Nottingham are developing two state-of-the-art techniques to study what happens to the faulty RNA in myotonic dystrophy after treatment with potential therapies. This could help to identify effective potential treatments that target the underlying molecular causes of myotonic dystrophy.

Follow this link for more information on the project:

100% funded by The Fight Fund!

Drug trial to treat Congenital and Juvenile onset Myotonic Dystrophy - RECRUITING NOW!

The study is recruiting participants from the UK who are between the ages of 16 and 45 and have congenital or juvenile-onset myotonic dystrophy type 1 i.e. experienced symptoms before the age of 12.

More information can be found on

We are OVER THE MOON to announce the FIRST drug trial for adults with Congenital/juvenile DM.

It is taking place at the John Walton Muscular Dystrophy Research Centre in Newcastle, UK, and is sponsored by the pharmaceutical company Amo Pharma.

The purpose of this study is to investigate the safety of a drug called tideglusib (AMO-02). Tideglusib inhibits an enzyme called glycogen synthase kinase 3ß (GSK3ß), which is over-active in people with myotonic dystrophy type 1. Preclinical research has shown that inhibiting GSK3ß can increase muscle strength and decrease myotonia.

In addition to safety, the study will also investigate the efficacy (i.e. potential benefits) and the pharmacokinetics (blood levels across time) of tideglusib.

The study is recruiting participants from the UK who are between the ages of 16 and 45 and have congenital or juvenile-onset myotonic dystrophy type 1 i.e. experienced symptoms before the age of 12. Participants must also be able to walk independently and meet a number of other criteria;

If you are interested in taking part in the trial, please get in touch with the trial sub-investigators: Dr. Nikoletta Nikolenko on 07870 517410 or email: or Dr. Tiago Gomes on 0191 241 8989 or email: