Preclinical research is a key step in the development of new disease treatments. Most frequently, a compound will only be tested in patients once it has been shown to be efficacious in an animal model that reliably reflects the human disease. Therefore, the choice of an appropriate animal model and the experimental parameters to assess the efficacy of a new chemical entity is crucial.
There are a large variety of animal models for both Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA), which often makes it difficult to compare results. These animal models may differ in the genetic background and the severity of the pathology, which can lead to differences in the response to the therapeutic intervention. This situation is aggravated by the multitude of experimental protocols that are used as efficacy readouts. As a consequence, parallel efforts cannot be satisfactorily compared.
TREAT-NMD aims to overcome this fragmentation by harmonizing the tools used in preclinical research. The goal is to identify a limited number of animal models and experimental protocols that are considered appropriate for use by research groups around the world, thereby improving the comparability of the preclinical work and accelerating the development of new drugs into the clinic.