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. "

Over 5 years they will run patient preference studies in both academic and industry settings. This is to provide a better understanding of what will be a recommended best-practice approach to patient-preference studies. This will show how patient preference studies can give valuable information to support decision making for regulators and HTA bodies.


The online Survey is NOW LIVE - To access the survey please click the link below:






If you’re interested in more information on this study, please get in touch with Dr Cecilia Jimenez Moreno at:   

or by calling 01912083105

 For more information, read the study information sheet

You can read more about the PREFER STUDY, here on the MDUK website

Visit the official IMI-PREFER page here





Focus Groups announcement.

Emma-Jayne from Cure DM CIC is delighted to be consulting with the Prefer project, and to announce the dates for the first set of Focus Groups (approx 1.5hours of your time)

If you are able to take part in one of these groups, please do – this is an important step to having our voices heard as patients and patient representatives, and to shape the future of studies, trials and treatments. If you feel you can’t take part in this step, please do contact Cecilia as there may be other ways you can help as the study moves forward. (Please mention Cure DM when you make contact)

As part of the IMI PREFER  project, Newcastle University is to conduct focus groups for people with myotonic dystrophy type 1 or mitochondrial disease, as well as their caregivers (including caregivers of children with Juvenile/Congenital onset DM1).

We want to know what people with these conditions think about different treatments (real treatments and hypothetical ones) and what trade-offs they are willing to make.  

A trade-off is the act of balancing a negative against a positive, for example the side-effects or risk of a treatment against the benefits.

This sort of information will help pharmaceutical companies to design more patient-centric treatments and clinical trials. So your input into this study is really valuable for shaping future research.

The focus groups will be about 1.5 hours long and will take place on:

·   Friday 15th March, London

·   Saturday 30th March – Nottingham

·   Saturday 6 April - Newcastle (In conjunction with MDUK research and   information day – Cure DM Pete and Emma will be here)

 £15 voucher will be given as a thank-you for taking part.


If you feel you would like to take part and support the Prefer project, but are unable to attend these meetings OR you are an affected patient with symptoms before the age of 20 – Please contact Cecilia, as you may be able to help on other parts of the study.