Hoping this letter finds everyone settling into the school year and getting ready for a little fall weather (and football). We are thinking often of our southeast friends (and several of our GP2C team and staff members) as they wait for Hurricane Dorian to pass. Please keep them in your thoughts and prayers.
We are also anticipating a fantastic program for Rett Syndrome Awareness Month coming in October (just around the corner)! Please stay tuned to GP2C.org, GP2C Facebook and our other social media pages. We will have daily posts for you to share with your community, and to help raise awareness and educate your followers. Rett Syndrome Awareness Month is also a great opportunity to fundraise for the cure! Our sites will include donation links throughout the month as we continue to #RiseAboveRett and support our research partners at Rett Syndrome Research Trust. If you are so inclined, please consider sponsoring a personal Facebook Fundraiser… we have all the tools and guidance here, and it only takes a minute!
From the research world…
We are all disappointed with recent news regarding delays in clinical trials for the Novartis/AveXis AVXS-201 gene therapy treatment. While the delay is unfortunate, the safety and protection of our loved ones is of utmost importance. You can read more about the status here, but I implore all to stay positive and optimistic that the trials will come. From the AveXis press release:
“Due to the data integrity concerns related to a specific animal testing procedure used in the development of Zolgensma and included in the application, we have decided to also review data quality and compliance with the preclinical work performed for AVXS-201, our Rett Syndrome gene therapy candidate. Out of caution, and to ensure that we have a robust data package for the FDA, we have chosen to repeat and add additional pivotal preclinical studies as well as new quality controls. Once these studies are completed, we will submit the revised IND (investigational new drug application) to the FDA, with the goal of rapidly progressing to clinical trials in Rett Syndrome patients. We expect that conducting these additional studies and completing the IND will take until the middle of 2020…. We recognize that this news will cause concern and disappointment. Please know that AveXis remains focused and steadfast in our commitment to the Rett program and to ensuring the highest levels of transparency and integrity with the patients and providers we serve, and health agencies.
As we have said many times, “keeping them ready” is the most important thing we can do today for our affected family members. At GP2C, we will continue our mission to support the Rett Syndrome community with daily encouragement, resources, and Rett University education programs to help our loved ones achieve to their fullest potential.
ICYMI… I highly recommend review of this June 5, 2019 article, Making Sense of it All, which plainly identifies the process (and challenges) for bringing new treatments to fruition. Thanks for all that you do. Our community is second-to-none with respect to supporting each other, and we will continue steadfastly until the cure is here.
Have a great September!