Research Strategy

The objective of the research strategy is to increase understanding of muscular dystrophy and allied disorders and to support research into and evaluation of potential management and treatment strategies

Specifically the research strategies are to:

1. To further understanding of the basis of normal and abnormal muscle function with the long-term aim of identifying candidate treatments or management strategies for muscular dystrophies
  • Progress basic research to have a sufficient understanding of muscular dystrophies and appropriate therapeutic targets with the long-term aim of generating candidate treatments

  • Appropriately promote the availability of MDC research grants. Encourage the establishment of consortia and/or collaborations (both nationally and internationally) to enhance the translation of research to the clinic

  • Scientific advances of the past decade have opened up the opportunity of exploring new options for the potential treatment of muscular dystrophies. Research into conditions caused by “loss-of-function” mutations will be focussed on developing innovative technologies particularly those with the potential to treat a range of muscular dystrophies:
    • Developing pharmacological interventions (IGFs, myostatin antagonists)

    • Replacing the affected muscle cells (cell transplantation therapies)

    • Restoring a wild-type copy of the gene (gene therapy)

    • Obviating the effect of the mutation (exon skipping)

    • Bypassing the mutation (e.g. upregulate utrophin in DMD)
    The potential of these approaches will be evaluated on a regular basis
  • Find out what members and potential members, as well as clinicians, regard as current unmet medical needs and aim to establish research proposals in these areas

  • Identify marketed treatments which may have therapeutic potential in muscular dystrophies either as treatments or to meet current unmet medical needs of patients

2. To fast-track promising treatments when they are close to clinical trials to ensure a rapid transition from the laboratory into the clinic
  • Register treatments for fast-track clinical development (e.g., orphan drug designations) where appropriate, and lobby to make the process more straightforward

3. To progress safe and effective treatments through clinical development to the marketplace in the shortest time
Clinical trials provide the evidence on which treatment decisions are based. Whilst providing funding for large scale clinical trials is currently beyond the scope of the MDC, the MDC is keen to support pilot studies, and work in partnership with other research funders and professional associations, and the pharmaceutical industry to support clinical trials.
  • Ensure there are appropriate Clinical Research Centres and facilities including databases of patients in advance of full clinical development

  • Work with scientists involved in a wide range of research and care to design innovative clinical research and development strategies for new management approaches and treatments

  • Develop close relationships with regulatory authorities to gain an understanding of their requirements for registration in this area


How the Muscular Dystrophy Campaign funds research
The Muscular Dystrophy Campaign funds the following categories of research:
1. Research grants
Project grants are funded for a period of up to 3 years. Project grants can include staff costs (excluding those of the principal applicant), consumables and equipment costs.
2. Service grants
  • Muscle Centre grants provide funding for centres of excellence enabling those affected by muscular dystrophy and related conditions to receive a range of expert services. They also fund clinical research fellows.

  • Muscle Network grants fund links between professionals to enable full access to expert advice and information on muscular dystrophy and related conditions.

3. Paul Walker Fellowships
This award is given on a biannual basis and is split between two Young Investigator Awards consisting of £2000 for a student registered on a PhD or MD programme and £3000 for a postdoctoral researcher within five years of completing a PhD. The fellowship is awarded to encourage new researchers in the area of muscular dystrophy and may be put towards, for example, travelling expenses for conferences and personal computer equipment.

Who the Muscular Dystrophy Campaign funds and where the charity funds research
The Muscular Dystrophy Campaign funds both PhD and postdoctoral scientists and clinicians in the UK engaged in research into at least one of the more than sixty different forms of muscular dystrophy and allied conditions that the charity covers. For a full listing of the conditions funded by the Muscular Dystrophy Campaign see Neuromuscular conditions covered by the Muscular Dystrophy Campaign. The Principal Investigator of a Muscular Dystrophy Campaign research grant must hold a contract, which extends beyond the duration of the grant, at an Institution approved by the charity. Collaborations are favourably viewed especially between researchers and clinicians but also with companies.

When funding is made available
There is only one application round per year. The application process begins with the submission of a two page outline in October. These are then triaged by the Medical Research Committee, and the applicants who are successful are invited to complete a full application form. The deadline for these is usually the middle to end of January. The applications are then sent to experts world-wide for peer-review and finally considered by the Medical Research Committee in the spring. The results are made available in June/July for funding to commence the following October.

The number of grants funded each year varies depending on the amount of free monies available for the year. The Muscular Dystrophy Campaign endeavours to fund as many projects as possible in order to maximise the potential of the grant portfolio.

New approaches for consideration
1. Maintain current process of inviting standard research grants on a yearly basis. Also invite applications for high-risk research. One or two research projects into innovative new approaches could be awarded with a limit of around £30-40K for one year only.
2. Strategy ‘think-tank’ to identify key areas of research which the Muscular Dystrophy Campaign is not currently involved in, but which could have a major impact on developing a treatment or clinical management of a condition. The Muscular Dystrophy Campaign could then put out a call for proposals in this area. These would be reviewed by a sub-committee of the Medical Research Committee. Fundraising would then endeavour to raise funds for MRC-approved projects, and if successful, the research would be funded.



Applying for a grant

The Muscular Dystrophy Campaign is currently accepting proposals for the 2007 Grant Round.

Apply for a research grant