Jennifer Handt

$SRPT & #DMD patients are under attack! ICER, representing the voice of payors, argues that the juice (successful patient outcomes) isn’t worth the squeeze (the cost)!!! Elevidys doesn’t represent a free pass for bad science. Several therapies that did not hit statistical benchmarks of benefit in randomized trials have become first in class treatments or part of a standard of care regimen because of actual clinical benefit. That includes Galafold for Fabry Disease and Danyelza for neuroblastoma. AT the end: The FDA mustn’t allow itself to be bullied by payors, politicians, pundits, or ill-informed academics. Most importantly, the FDA mustn’t be inappropriately restrained by the shackles of the status quo. H/T @JonU235 for unlimited supply of reports! realclearhealth.com/blog/202…

$SRPT “awaiting drugs with efficacy levels high enough to satisfy JAMA writers may never materialize” However, it’s essential for JAMA to step out of its ivory tower and delve into the practical realities. Specifically, they should closely examine the groundbreaking work being done by #Elevidys. In the past, it took a practical clinician like Janet Woodcock to recognize the utility of #Eteplirsen that is now demonstrating an incredible survival advantage. Without her foresight, these young patients would still be trapped in a bleak and discouraging limbo. These days Dr. Marks is recognizing the real advantage of $SRPT's gene-therapy! He understand that NSAA is not the right test to assess #DMD post GTx but requires a longer-term than 52Wks... A longer time is well needed. On the other hand, endpoints like TTR & 10MW play a crucial role in demonstrating the real-life impact on #DMD patients after gene therapy. Let’s continue to advocate for advancements that benefit patients and improve their quality of life.

For all the naysayers & for the JAMA's writer who is far from understanding what is really #DMD & how it affects the kids lives & their family members. $SRPT's GTx hit stat sig. all pre-sepcified key secondary endpoints, TTR & 10MW If anyone don't understand what is TTR (Time to Rise from floor) just watch this short video taken by Jennifer, the mother of #DMD kid who had #ELEVIDYS gene-therapy & amazed with his progress & strength!