Preclinical research

The preclinical phase of research is so critical to the decisions made about a possible future therapy hence, it is very important that experiments done at this stage are based on best practice. This means both choosing the most appropriate animal model and doing the experiments in a way that ensures they are comparable and reproducible in different labs. This minimizes the risk of discarding a useful therapy, or conversely, taking a compound into trial in humans that is then proven to have no effect. The TREAT-NMD website hosts documentation covering a number of standardised protocols and procedures that are widely used in with disease models.

Duchenne muscular dystrophy

MDX and GRMD disease models

Spinal muscular atrophy

SMA models

Congenital muscular dystrophy

CMD models

Please note – Currently, these operating procedures, although extensively used worldwide, haven’t been endorsed by EURO-NMD. They are included in this section of the website to highlight some of the work that has already been undertaken, as such this should not be considered a comprehensive list of procedures for disease models.