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| Sponsor | Imperial College London |
| Funder | Department of Health (DoH) |
| CI | Professor Francesco Muntoni |
| sites | London GOSH |
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More information |
Background
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).
Recruitment
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]