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Testimony before Congress on Accelerated Approval by mom with the twins in the clinical trialThis is just a partial post....please read the link in its entirety as it is an excellent piece.
"Perhaps most encouraging is the news from Sarepta that its candidate therapy, Eteplirsen, had produced statistically significant levels of Dystrophin in patients participating in the Phase IIb clinical trial. In addition to showing increased levels of Dystrophin, the study also found a significant clinical improvement as measured by the 6-minute walk test standard.
Both Jack and Nolan participated in this trial. I would love to discuss the details of our experiences during the trial with anyone who is interested after this briefing. The main point I would like to drive home about Eteplirsen is that there is a finite window of opportunity here for these boys. For maximum benefit, it is imperative that these boys receive the drug as early as deemed necessary. As illustrated quite clearly by my own sons' results at the end of the first 24 weeks, there is a tipping point before which Eteplirsen should be dosed. Although Jack and Nolan qualified without issue for this trial, they showed the most deterioration from the start, therefore; their loss of ambulation during the trial came to no great surprise. The progress that the other 10 participants are achieving is phenomenal. They continue to progress to this day. As you can imagine, this news has left many of us in the Duchenne campaign quite eager and optimistic, particularly as Sarepta prepares to move forward as quickly as possible in submitting its data to the FDA, which brings us right back to the reforms Congress included in the FDASIA law. While the ball is presently in Sarepta's court as it prepares for the filing, once their application arrives at FDA, it is critical that it be reviewed as expeditiously as possible because no alternative treatments or therapies exist and the disease is 100% fatal. The drug should therefore qualify as a breakthrough therapy. Our community is counting on FDA's "patient-focused drug development" initiative to move these interests forward. In addition to prompt review, we trust FDA will take into account the views of patients and/or their parents/guardians, in the case of minors, particularly when it comes to the all-important issue of assessing levels of benefit and risk offered by a therapy."
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