CMT-NET is an interdisciplinary German network with the intention of bringing together experts from different areas of research to collaborate on the molecular pathogenesis, epidemiology, diagnosis, and treatment of CMT. Key questions of CMT NET are:
1) What are genetic and acquired risk factors of CMT1A patients determining disease severity?
2) What is the disease burden of CMT patients in Germany at different disease stages?
3) What are therapeutic options that can easily be translated from animal models to patients?
4) How can we measure disease severity and progression in CMT patients in order to ensure trial readiness in CMT1A? Therefore, CMT-NET aims
(i) to examine mechanisms of the disturbed axon-glia-interaction and neuronal vulnerability in CMT1A using transgenic fly, chick, mouse and rat approaches
(ii) to identify genetic modifiers and non-genetic risk factors in CMT
(iii) to identify and validate novel targets to rationally design pre-clinical therapeutic approaches in CMT,
(iv) to identify and validate outcome measures in children and adults with CMT, including biomarkers from blood,
(v) to establish a skin and nerve biopsy biobank for CMT patients,
(vi) to establish an internationally harmonised CMT patient registry.
