DMD+Discussion

Discussion:

While the FDA has approved Eteplirsen there are issues with the clinical studies when it comes to sample size. Both major studies that were reviewed acknowledge this issue. Due to the critical nature of the disease, this was overlooked. There has also been criticism that the statistical significance for efficacy is border line and barely meets requirement standards as well as the high cost of treatment (upwards of $300,000 a year). There are differing thoughts on the issue. Some researchers feel that emotional testimony from parents of affected DMD patients drove the decision to accelerate FDA approval without the typically robust experimentation history normally required for drug approval. Others believe this was necessary to expand treatment options for those suffering from DMD.

Abstract Materials and Methods Results (back)

Broader Impact (next) Works Cited