New failure of anti-myostatin therapy in DMD

The investigators who conducted three clinical trials to test the efficacy and safety of taldefgrobep alpha (a myostatin-inhibiting molecule) in Duchenne muscular dystrophy (DMD) publish disappointing results in a single article:

  • this was a phase I trial in healthy volunteers initiated back in 2014, followed by two phase Ib/II and II/III trials, all involving 180 children with DMD,
  • while biologically, myostatin suppression and a variable increase in muscle volume were demonstrated, efficacy in terms of strength was not,
  • however, the product was well tolerated.

This work underlines, once again, the limits of the anti-myostatin approach in DMD.


Taldefgrobep Alfa Study Group. The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy. Muntoni F, Byrne BJ, McMillan HJ et al. Neurol Ther. 2024 Feb.