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My eight year old just got diagnosed with DMD last month. We thought that’s what it was and the genetic test confirmed two days before we went to Seattle for the super muscle clinic. We have not started any medication as of yet. The doctors wanted to give us a chance to research and see what we wanted to do as a plan of action. One of the doctors told us about a family who had a RUclips channel with DMD who had the gene transfer therapy. I searched until I found your channel and have been watching some videos. Great information. DMD does not run in my wife’s family so we were totally blindsided by the diagnosis and we have two older boys that do not have DMD. We live in Fairbanks, Alaska and there is no centers that treat DMD here. I believe I understood you saying it has been almost a year since the gene therapy for Mason. Have you seen any positive/negative results or concerns in this procedure? I have also been looking at the Exxon skipping. Thanks, Jackie

Brilliant I’ll have to try it. Much less of a mess

Thank you so much for your thoughtful video!

Sounds like a good alternative. Good luck

It would be very very nice to see a comparative video about Mason's muscle-based abilities before he was treated with Elevidys and nowadays.. 🙂

He is such a funny little dude! 😂

So cute ☺️

happy birthday🎉🎉🎉

Happy Birthday to Mason! His muscular abilities are really awsome, great.

Happy birthday to you mason🎉

Happy 6th birthday Mason!🎉🥳

Was the MRI to check on his heart?

We have our eye apt with Cleveland Clinic next week for same issue.

Why don’t you want to stop the use of steroids? I’ve seen presentations by specialists from Sarepta, and they mentioned that steroids may not be necessary. We are planning to stop the steroids in a few months. Our son received gene therapy only two months ago. So, we plan to use the steroid for another 3-5 months.

Our 5 year old DMD son also was diagnosed with autism. Very common as we’ve come to find out. As for the steroids and eyes this is news to us. We are starting Vamorolone in Dec. I too have a difficult time pronouncing the word with our Neuro team. Great video! So happy that you post it gives some normalcy to our life.

You guys are doing great. I don't know if I ever really grieved after my daughter's diagnosis but my wife sure did. I jumped right into the learning about the disease part. I was able to garner some hope amid all the bad stuff. You guys are the ones that know your kids so trust your instincts. Research recommendations from doctors and therapists. Most therapies whether physical or drug have pros and cons. It looks to me like you guys are doing great with this. I am very confident in the gene therapy. I've been following the development for 8 years now. Watching your journey helps me understand what it's going to be like when my daughter can get it hopefully.

My daughter has limb girdle muscular dystrophy type 2C with duchenne like progression. It's not exactly the same but close. We ended up getting her a psychologist rather than trying any ADHD drugs. That was 8 or 9 years ago and she still meets with the psychologist every other week. It's been one of the best things we ever did. When she was Mason's age she had behaviors that could have been ADHD, in fact that was what the first neurologist we saw thought she had. That neurologist prescribed Adderall. I looked up the side effects and saw heart issues. My gut told me I shouldn't be giving her that without seeing some other doctors first. Eventually got the LGMD2C diagnosis from another neurologist. The ADHD like symptoms were from anxiety most likely because she knew she was different than her peers and not able to do the things they could. Another thing that helped immensely was vision therapy, not sure if it would help cataracts but it sure has helped with school stuff over the years. I really appreciate you guys sharing your journey. We've been "patiently" waiting for gene therapy for LGMD2C for about 8 years now.

Its interesting to see that the behavior issues are common with DMD i did not know that. Im having alot of these struggles too!

My 15 year old is on Deflazacort and we were told he has Cataracts 3 months ago. Unknown side effect of the Exon skipping drug?

Thanks for giving us those updates, it helps us getting though our journey too. For the Vamorolone (Agamree), my son is on it since august 2023. He has no side effects and is growing normally. It amazed the neurologist since it's the first kid that she follows that has it. So, if you have the change to switch, I would do it in a heartbeat... I talked to the father of an older kid (±17) and they fear that if he changes at that age, bones and muscles won't "grow" at the same pace and other problems might occur. Our sons are facing so much, if we can save them a little trouble...

Thanks a lot for sharing such an important quote❤ I hope faith will help us all to go through the difficulties 🙏

Are they on Deflazacort?

Omg This made me laugh! What did we end up with?

😂😂😂

That sounds delicious! Do you have a recipe to share bychance?

I would love the recipe, please. Thanks.

Do I need to buy you a muffin pan?? lol

Do I need to buy you a muffin pan?? Lol

Hello, could you tell me what is your boy's Creatine Kinase level a few months after gene therapy? Thank you!

Masons smile is literally the best ❤

Hello, We received Elevidis 6 weeks ago. What is your son's Creatine Kinase level? Thanks!

It was awesome to meet you and your family! I enjoyed making friends who understand this disability. It's cool to be on RUclips!

I’m a carrier too.

Aww this is such a great video!!❤

Did he have a loss of appetite at all after receiving the gene therapy?

Backpack and a lunch box and a water bottle

Wow, that's a real miracle, Mason does the stairs like every "normal" boy at his age. Asesome...

He this not have room to sleep in his bed he have a lot toys on his you need to blow your nose do you have a cold are a running nose what is dmd stand for bed animal toys how old is your boys what grade in school how did he broke his foot did he get a x-ray

My kid only wears afo’s during the night.

Jack's face on the log ride 🤣🤣🤣

Sorry should have watched the whole video before commenting. These are wonderful shoes, the kids can do it themselves!

Look into BILLY shoes

im 19 and have high functioning autism, i STRUGGLED to get das. i did the call with my behavioral therapist, 2 healthcare professions said no so i had to get a coordinator on who got a 3rd healthcare professional that finally said yes. the entire call took over an hour ( 5 minutes waiting in the queue ). it was very stressfull for me and i love disney so much but the thought of having to do that again really upsets me, maybe it will be easier next time? so glad you got it though, DAS really does help and do wonders! i hope yall had fun :)

What is the name of the facility in PA?

I highly recommend a port. Drew used to have the worst 2 yr old anxiety and fear, and screaming. The port makes it so much easier and no crying

Inspiring! So much love in this family. Please provide an update on weekend dosing. We are looking at starting weekend dosing for our 5 year old. Blessings to you and your family.

I live in the UK and we are going to Disney World and Universal Studios in 9 days time! I have primary progressive multiple Sclerosis and have had it for 11yrs, about 5 yrs ago when we last went to Disney world DAS pass was so much easier, but we will be trying to contact Disney World to see if we can get it! I’m in a wheelchair 90%of my time and my bladder can wake up at anytime and say I need to go now! And I don’t have long to hang around! Hopefully we will get in??

Emflaza?

What is afo stand for

Bryan's Ribs ™️ 🤣🙌🏻