LTBP4

Targeting muscle inflammation and fibrosis

With the funds raised in 2016, the DRF is tackling inflammation and fibrosis, which are two key problems for Duchenne boys. It is believed that the protein LTBP4 prevents the activation of a key biological pathway (called TGF-beta) that is an important contributor to these disease factors. In a natural history study of Duchenne patients, the presence of a modified version of this LTBP4 protein correlated with patients’ ability to walk until later ages.

Based on studies in the lab of Dr Beth McNally, currently at Northwestern University (Chicago), DRF is helping fund a potential therapy that aims to stabilise the LTBP4 protein to reduce signs of muscle disease and increase overall function. The project is being managed by Solid Biosciences.

Pre-clinical research

£70,000 contribution

Funded in 2016-17

Chicago, USA

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 to gather further insights into the prevention and/or treatment of muscle fibrosis in boys with Duchenne. Development of therapeutic strategies that will tackle fibrosis will not only enhance the chances of success for treatments like gene therapy, but will also ensure that as many boys as possible will be able to benefit from these promising developments. The newly funded projects supported by the DRF will address those shortcomings and I am confident that the successful completion of those will make seminal contributions to the field of Duchenne.

Professor Ronald Cohn

Head of the DRF Scientific Advisory Board; Chair, Department of Paediatrics, University of Toronto; Paediatrician-in-Chief, The Hospital for Sick Children, Toronto

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