For a complete understanding of the role of the hydrophobic core within Pip peptides, one would ideally wish to study the activities, pharmacology and biophysical parameters of a much larger range of sequence-variant Pip-PMO conjugates. The need for multi-mg synthesis of each conjugate as well as the availability of a large number of mdx 37 ). This is greatly promising for the Pip6-PMOs, as it would not be considered the optimal peptide yet still demonstrated the significant therapeutic effect of this group of peptides. These new leads provide a good basis for identification of a Pip-PMO candidate suitable for detailed physiological studies of muscle and heart function, as well as thorough toxicity profiling including dose escalation studies, in anticipation that one such Pip-PMO will proceed to clinical trial.