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" ... "The FDA was very direct in collaborative on its feedback," says Douglas Ingram, Sarepta's chief executive, "and we can rely on the precedent that Exondys levels of dystrophin are reasonably likely to result in benefit." ... "
" ... One thing’s for certain: the data live up to the heady expectations of Sarepta’s investors. Shares in the company have run up 80% so far this day, giving the Cambridge, Massachusetts-based company a market capitalization of $6.9 billion. Investors are likely to be focused on a measure of whether a test called the western blot shows that the gene therapy has sufficiently raised levels of dystrophin, the protein that is dysfunctional or missing in Duchenne patients, and without which the body cannot build and maintain muscle. ... "
" ... Sarepta’s gene therapy uses a virus, a modified version of the adeno-associated virus, to insert a shortened version of the gene for a protein called dystrophin into patients’ cells. Dystrophin is missing in Duchenne patients, causing them to lose muscle tone, forcing them into wheelchairs, and killing them, often by early adulthood. ... "