The development of an animal model of giant axon neuropathy using zebrafish, which reproduces the loss of mobility observed in patients suffering from the disease, has led to the identification of new drug candidates. The results of this French study, supported by AFM-Téléthon, were published in May 2023:
- Pharmacological screening to identify small molecules capable of restoring both physiological and cellular deficits in the animal model.
- High-resolution behavioural, computational and imaging analyses were carried out to refine the results, enabling the researchers to select five drug candidates.
- These molecules restore locomotion and axonal growth and stabilise neuromuscular junctions in the zebrafish model.
- They could be integrated into a repositioning approach to accelerate the development of a treatment for this neuropathy.
