This quote is not mine but I love it.
I am going to steal it for every single mitochondrial presentation. For our doctors, for our researchers, for our government, for you….please…..be mighty….be bold……we are waiting.
Friday found me here:
I invite you to read the caveat on the bottom of the poster ‘Given the complexities of primary mitochondrial diseases, and by extension the difficulty of designing informative clinical trials in this group of rare diseases, FDA is organizing a scientific symposium with the goal of bringing together academic physicians, FDA regulatory experts, patient advocates, and other interested stakeholders to exchange ideas and advance drug development in this challenging field.’
When the FDA puts this in print, our call to be mighty is mighty.
I pulled up on Friday in my Uber…..just a simple English major from Colorado…… with our super-smart Mito experts and the frickin’ FDA.
Well butter my biscuit and call me for breakfast.
Here’s the skinny; you Mito folks we have an audience.
We have an ear of the FDA.
Thank you to the UMDF, Stealth BioTherapeutics, MitoAction, MDA, everyone in this space, thank you for promoting our ear.
Clinical trials within the mitochondrial space are not easy. Mitochondrial diseases spread among 37 MtDNA genes and over 250 nuclear. Our family carries a mutated POLG-1 gene, which is a nuclear inheritance but the presentation even among our family is different.
Every night Hubs would rub Sammers head and say ‘good night, Samantha, keep fighting the good fight.’
We lost that fight.
I hate that we lost that fight.
When the FDA started talking about Pediatric testing and the ethic barriers to certain tests, I got up and talked. I talked about when Samantha was diagnosed with Infantile Spasms; when her poor precious brain was seizing 90% of the day and the only med that could help help her was Vigabatrin. And when I found out Vigabatrin was not FDA approved because it was known to inhibit peripheral vision.
I didn’t give two poops about her peripheral vision. Stop the seizures.
Stop the seizures.
Stop the seizures.
We paid out of pocket from Canada for Vigabatrin and for a brief moment, I lived the life of a Vigabatrin drug lord because it was approved in the States.
The FDA needed to know my life.
The FDA needs to know your life.
They need to know that you would sacrifice peripheral vision to stop the seizures. That your life is so far from normal, you would give up what seems normal for a bit of normal.
My challenge to you Mitochondrial community.
Please be Mighty.
Lets talk about our story. We talk about a Natural History study, mapping out genetics and physiology. What about a person study? Who were you before your mitochondrial disease? Before your child’s mitochondrial disease? We now have an ear to the FDA.
Be fearless, be bold, know you have a voice.
We have nothing to lose.
I will take your mitochondrial story: firstname.lastname@example.org