Lower Steroid Doses! A Win for Mason's Health

GREAT NEWS! Mason is finally able to decrease his dosage of Prednisone! Join us as we delve deeper into this positive development and other updates for the Bafus family. Jada, as a carrier of Duchenne muscular dystrophy, gets an Echogram on her heart, and we also learn that Mason will need to continue labs for a few more months. Also Happy Mother's Day to all the Moms out there!
Disclaimer: This video is for informational purposes only and should not be a substitute for professional medical advice. Always consult with a doctor about your specific health concerns. These videos simply serve as documentation surrounding this treatment and decisions we've made, as parents, to potentially better the lives of our children.
====================
What's Elevidys? Elevidys infusion is a gene therapy from Sarepta. It's the first gene therapy FDA approved for pediatric patients (4-5 years old) with Duchenne muscular dystrophy.
HUGE THANK YOU to Sarepta (Elevidys) and the Seattle Children's Hospital!!!
====================
Hey! If we're meeting for the first time- We are the Bafus family, and we are starting this RUclips channel to give hope, document our journey, and to shine light on duchenne muscular dystrophy.
For our first 7 days, we posted one video a day, documenting Mason's $3.2 million, once in a lifetime gene therapy.
You can watch that here ➡: • Mason's Gene Therapy
====================
We are typically private people, just like you, but this disease has forced us more into the public, and that's ok. We've learned that this is not a disease that anyone can fight alone. So we decided to take to RUclips to help spread the message and raise awareness of this rare disease.
You can follow us here 👇
/ bafusfamily
/ @bafusfamily
#duchenne #duchennemusculardystrophy #dmd #musculardystrophy #familyvlog

[@JadaBafus]

I hope my fellow DMD moms had a wonderful Mother’s Day! ❤

[@ShahNawaz-kp3jn]

Hi Madam My three disabled brothers suffering from Disease Muscular Dystrophy I'm from Pakistan

[@jaysimoes3705]

Good. The less prednisone the better (to some extend). Have you alreayd considered Vamorolone? I am unsure if it is available alreay in the USA? How about Givinostat? I think it is not available yet, but Vamorolone has less side effects (better for bones I think and much better for his growth, become normal again!) and givinostat is I think an HDAC inhibitor, which means it treats Duchenne very differently than Elevidys and in all likelihood should be able to combine with it without side-effects (but that is my guess).
Good to see the other boy can get some relief with exonskipping! All the best and keep up the good work. And indeed: eat healthy, it is so so so important also for the parents. At least that is what I think.

[@BafusFamily]

We have looked into these options briefly! Our neurologist and medical team doesn’t want to change up the steroids quite yet this soon post the gene therapy. We plan to have our other little guy on one of these when the time comes! But our goal is definitely to not have them on the steroids! Also we plan to try weekend dosing as well. We are excited for the Econ skipping to start! Thank you for the advice! 😊

[@jaysimoes3705]

@@BafusFamily I can understand that. There is one example of two kids getting Galgt2 therapy (briefly a therapy that has nothing to do with dystrophin, but it changes a thing elsewhere and this thing uses utrophin to stabilse the muscle membrane). One kept on using steroids and did pretty well, just not as good as kids on elevidys. The other kids parents stopped steroids and he became non-ambulant soon after. I think this boy was already not doing too well and it is just one boy, so we can't connect it to taking away the steroids. But it does go against the advice of doctors.
Good luck with the skipping! I hope it all works out fine. I do wonder with the older one who has had Elevidys: do you see clear improvement? A changed course of the disease? Take care!