Non-invasive imaging biomarkerReducing biopsies and improving dystrophin analysis
Biopsies have become a difficult necessity for Duchenne patients who are involved in clinical trials. In addition to the stress on patients, each muscle can present disease differently, making certain samples hard or inappropriate to analyse.
With the funds raised in 2016, DRF is helping fund the development of a platform technology that utilises classic imaging techniques to enable clinicians to measure changes in dystrophin expression in Duchenne patients without biopsies. These funds have enabled Solid Biosciences to form key collaborations with academic and biotechnology partners to perform necessary early development activities. If successful, this technology could be used as part of future clinical studies to evaluate disease correcting approaches.
Funded in 2016-17
Cambridge, MA, USA
What is a biomarker?
In medicine, a biomarker is a biological feature that can be used to measure the presence or progress of a particular disease. Biomarkers can be used to assess the effectiveness of a particular treatment in helping to alleviate the effects of a disease. We are helping fund development of new biomarkers for Duchenne that aim to monitor the progression of Duchenne more effectively and measure patients’ response to new treatments in clinical trials.
Why are we supporting this project?
Despite the significant acceleration of therapeutic developments in the field of Duchenne in the last few years, a number of issues remain to be solved and require dedicated support and attention. We need better and more reliable outcome measures for boys with Duchenne, which is an area that has been insufficiently explored in the field so far. Characterisation of better outcome measures will enable the field to monitor disease progression and benefits of any therapeutic strategies in a more coherent and reliable manner. The newly funded projects supported by the Duchenne Research Fund will address those shortcomings and I am confident that the successful completion of those will make seminal contributions to the field of Duchenne.Dr Ronald Cohn
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