A substantial number of patients with neuromuscular disease—about 45 percent—could see beneficiary effects from a new cocktail drug recently covered in the journal Proceedings of the National Academy of Sciences.
The new treatment was developed by experts at the University of Alberta.
The condition of focus was Duchenne muscular dystrophy (DMD) and the treatment developed could bring about an effective way to reduce symptoms among patients.
“Most patients have out-of-frame deletions in the DMD gene, leading to dystrophin absence in muscle. There is no cure for DMD, but exon skipping is emerging as a potential therapy that uses antisense oligonucleotides to convert out-of-frame to in-frame mutations, enabling the production of truncated, partially functional dystrophin,” according to the study.
“Currently approved exon skipping therapies, however, have limited applicability and efficacy.”
“Here, we developed a more economical approach to skip DMD exons 45 to 55 (a strategy that could treat nearly half of all DMD patients) and identified DG9 peptide conjugation as a powerful way to improve exon skipping efficiencies in vivo,” the study also states.
Researchers combined the antisense oligonucleotides with a new peptide, a short chain of amino acids, as part of its newly developed treatment.
“This study provides proof of concept toward the development of a more economical and effective exons 45 to 55–skipping DMD therapy,” the authors concluded.
