How to trigger regulatory change?

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Jon Moulton
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How to trigger regulatory change?

What is the appropriate response when a technology exists that can ameliorate an otherwise certainly fatal degenerative disease, but the drugs cannot be developed under current regulatory structures?  This is the problem posed by exon-skipping oligos targeting Duchenne muscular dystrophy.  Though a single sequence of a Morpholino oligo has completed a phase 1 trial and shown efficacy in restoring dystrophin expression in localized muscle injections, the road ahead is expensive to approval and must be completed seperately for each sequence needed.  Different patients have mutations at different locations in the huge dystrophin gene, and will need different oligo sequences for treatment.
A more detailed discussion and links to a 2009 paper describing the regulatory barriers is in another section of Scientist Solutions: