Queen Square Centre for Neuromuscular Diseases


Restoring dystrophin expression in Duchenne Muscular Dystrophy: a phase I/II clinical trial using AVI-4658

Sponsor Imperial College London
Funder Department of Health (DoH)
CI Professor Francesco Muntoni
sites London GOSH
More information


The faulty protein in >70% of DMD is dystrophin. Antisense therapy has the potential to restore effectively its production. This could increase life expectancy through improved muscle survival and function. Recent research has shown the potential of this technique to

  • skip mutated dystrophin exons
  • restore the reading frame
  • generate functional dystrophin protein

We have shown proof-of-principle in human cell culture and animal model studies. We now wish to use this approach to induce dystrophin exon skipping in children with DMD.

Trial information

Primary objective: to assess efficacy and safety of intramuscular administered morpholino oligomer directed against exon 51 (AVI - 4658 PMO).


Children with DMD older than 10 years with mutations than can be corrected by the skipping of exon 51 [45-50; 47-50; 48-50; 49-50; 50; 52; 52-63]