DM2: targeting mechanisms disrupted by marketed molecules

  • Two articles published in the last 12 months present new therapeutic avenues in myotonic dystrophy type 2 (DM2) with encouraging results in Drosophila models of the disease.
  •  On the one hand, a screening of 3140 molecules on the market has identified 10 of them for their benefits on the survival and motor capacities of flies suffering from DM2. It should be noted that 4 of these molecules act on the activin signalling pathway.
  • In addition, polyamine supplementation restored the motor capacities of the diseased flies. The ZNF9 protein, mutated in DM2, is thought to be involved in the metabolism of polyamines and to contribute to muscle damage.


Translational control of polyamine metabolism by CNBP is required for Drosophila locomotor function. Coni S et al. Elife. 2021 Sep


Reducing the Excess Activin Signaling Rescues Muscle Degeneration in Myotonic Dystrophy Type 2 Drosophila Model. Deng J et al. J Pers Med. 2022 Mar.