CORDIS Project
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This project aims to develop a platform for accelerating drug development for neuromuscular disorders by utilizing patient-derived cells and advanced imaging techniques. It focuses on creating reliable methods to measure dystrophin levels in cell cultures, which will aid in the development of targeted therapies.
Neuromuscular disorders (NMDs) affect an estimated 300.000 people in Europe and no therapies are available for these highly incapacitating and lethal disorders.
In the last few years, this status quo has shifted dramatically with the appearance of new RNA therapies targeting several of these disorders.
Several clinical trials are currently underway but future development is being slowed due to several motives, mainly related to the requirement of personalised approaches for rarer mutations and t…
FUNDACION VASCA DE INNOVACION E INVESTIGACION SANITARIAS
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