Cure CMD and TREAT-NMD join forces to establish common protocols for efficacy studies in MDC1A preclinical research

It is now one year since Cure CMD and TREAT-NMD joined forces to facilitate and encourage preclinical research on a mouse model for MDC1A, which is caused by mutations in laminin α2 (also called merosin).

A group of researchers familiar with the animal model for MDC1A, the LAMA2dy-w mouse, responded enthusiastically to the call initiated by Cure CMD and TREAT-NMD and worked hard in the last months to establish standard protocols and guidelines for efficacy studies on this model.

The results of these efforts are several documents that will be useful for future preclinical research concerning MDC1A. One document contains recommendations on the care and handling of the mice with the aim to minimize the variability in the results obtained by different laboratories because of the different handling of this fragile mouse. Additionally, five standard operating procedures were elaborated for endpoint measurements commonly used to assess the pathology of mice. They cover the assessment of peripheral nerve pathology, of inflammatory responses and protocols to examine muscle histology (H&E stain, Massone’s trichrome stain, CSA, fiber number, centralized nuclei, Feret’s diameter and fibrosis). Three additional protocols are expected to be finalized in the next months, which cover measurements of spontaneous physical activity, grip strength and blood creatine kinase. The use of standard protocols is expected to reduce variability and encourage the implementation of new tests in the laboratories. Each of those documents is the result from the exchange of know-how and the sharing of methods between different laboratories, and therefore ensures objectivity and reproducibility. All finalized protocols are free for download here.

The LAMA2dy-w (now also called Lama2tm1Eeng/J) will soon be available at Jackson Laboratories as a colony. Orders can be already placed at

With the mice freely available, the recommendation on mouse care and the standard protocols, preclinical research on MDC1A will hopefully increase the use of this mouse model and improve the quality of preclinical studies aimed at determining treatment efficacy.

This progress would not have been possible without the effort of Valerie Allamand, Dean Burkin, Anne Connolly, Janice Dominov, Madeleine Durbeej-Hjalt, Eva Engvall, Mahasweta Girgenrath, Jeff Miller, Kanneboyina Nagaraju, Markus Rüegg, Anne Rutkowski, Volker Straub and Raffaella Willmann.

12 Apr 2017