If the numbers confuses anyone, there is visual evidence of the dystrophin production as well. Take a look at the slide below showing the progression of dystrophin production with eteplirsen. If you look at the 48-week biopsy slide and the dystrophin positive fibers, do you believe this looks closer to the normal healthy patient biopsy (on the far right) or does it look closer to an untreated DMD patient's muscle biopsy (on the far left)? I'll bet if you asked any five-year-old DMD patient which muscle biopsy looked closer to the 48-week eteplirsen biopsy, they would pick the normal non-DMD slide.
Eteplirsen doubters contend that only with a large clinical trial involving hundreds of DMD children will we be able to collect the data needed to satisfy the regulatory system. But let me tell you, during the face-to-face discussions I and other other DMD advocates had with the FDA (including Commissioner Margaret Hamburg) were were told repeatedly that the agency wanted to see smaller, innovative trial designs that targeted the underlying cause of the disease. FDA is aware of how quickly DMD patients progress and it doesn't want large-scale clinical trials for this disease. It was my sense that the FDA was willing to be flexible and eteplirsen might be the opportunity FDA was looking for to demonstrate their proactive demeanor in accelerated the approval of drugs for diseases like DMD.
At the end of the day, the data speak for itself. The natural history data of DMD patients is undeniable. Left untreated, those diagnosed with this disease follow a predictable decline in muscle function and walking ability. There are few outliers. The eteplirsen dystrophin data are real, the clinical benefit is evident.
Bolstering the case for the drug are anecdotal stories circulating in the DMD community. Eteplirsen patients in the Sarepta trial are beginning do to things physically they never could do before. That rarely, if ever, happens in DMD patients, particularly in this age group. These are important and measurable patient-reported outcomes where parents are seeing gains and stabilization in their DMD kids.These gains have an enormous impact across the board -- psychologically, sociologically, economically. They are measurable, important and something the DMD "experts" have not taken into consideration. Stopping or slowing the progression of DMD spares families from having to move to a one-story house, buying a handicap van, the fitting for a power-chair or installing ramps to their home. Instead, they are celebrating things like making a basket in basketball for the first time, participating in a 5k, trying on ice skates and jumping over a bike rack to impress a girl. My family is living with this horrible progressive disorder. We finally have the opportunity to impact this disease. How much further devastation and loss could result if this drug is not approved in the upcoming months? Far too many "experts" have lost sight of the bigger picture because they are lucky enough not to have to deal with DMD everyday of their lives. Let's be clear: DMD equals death and eteplirsen deserves accelerated approval now. Readers might think that I'm biased and overly emotional because I want this drug to work for my son. Actually, I'm capable of evaluating clinical trials just like anyone else, I have been doing this work for 12 years, I have evaluated many potential DMD treatments. I wouldn't be as passionate about eteplirsen if I wasn't convinced it works. I have seen the benefits of eteplirsen with my own skeptical eyes. Eteplirsen works. McSherry owns stock in Sarepta Therapeutics.
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