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...
Thank you do much for sharing your experience that helps us so much! I am so happy to hear that it's working well for your son! That makes me even more confident to make the switch now. Already got our first denial so the fight begins 💪🏼 and I agree anything we can do to make their lives that much easier 💙
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.
Thank you for your advice on this topic! I'm sorry for your daughter's diagnosis I looked it up and definitely a very similar progression. And I think you are spot on about lot of the ADHD symptoms easily could be a form of anxiety. I am diagnosed ADHD myself and I know for a fact it's anxiety based so that makes perfect sense! Trying some therapy may really be beneficial vs more pills with more side effects. That you for this suggestion and your experience! I'm going to look into this. Thank you for watching our journey it means a lot to us! What's the status on a gene therapy for your family? Is there one yet available or in the works?
So far the clinical trial by Sarepta is scheduled for early 2025. Still a chance she has antibodies though. She completed the 3 year natural history study in August this year so she is all ready when they are. We started going to Nationwide in Columbus to see Dr. Jerry Mendell in 2017. At that point the gene therapies he was getting started on were the limb girdle sarcoglycanopathies, 2D,2E and 2C. The virus they use is able to fit those entire genes so in theory much easier than duchenne. There's quite a few TH-cam videos with Dr. Mendell describing the evolution of gene therapy. He is a saint.
His name definitely is talked about we have heard of him at conferences :) i believe it! we are really praying they can expand this to other MDs sooner than later! it is amazing what they are finally coming up with.
It's so interesting to learn of the commonalities with DMD and other neurodevelopmental disorders like adhd! My guess is maybe due to the lack of dystrophin in the brain? The more we learn the more it starts to make sense!
I'm sorry to hear that! These side effects are brutal. In our case it's pretty atypical with him only being 5 and confirmed to be caused by his prednisone so I'm not sure if exon skipping can be a factor 🤔
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...
Thank you do much for sharing your experience that helps us so much! I am so happy to hear that it's working well for your son! That makes me even more confident to make the switch now. Already got our first denial so the fight begins 💪🏼 and I agree anything we can do to make their lives that much easier 💙
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.
Thank you for your advice on this topic! I'm sorry for your daughter's diagnosis I looked it up and definitely a very similar progression.
And I think you are spot on about lot of the ADHD symptoms easily could be a form of anxiety. I am diagnosed ADHD myself and I know for a fact it's anxiety based so that makes perfect sense! Trying some therapy may really be beneficial vs more pills with more side effects. That you for this suggestion and your experience! I'm going to look into this.
Thank you for watching our journey it means a lot to us! What's the status on a gene therapy for your family? Is there one yet available or in the works?
So far the clinical trial by Sarepta is scheduled for early 2025. Still a chance she has antibodies though. She completed the 3 year natural history study in August this year so she is all ready when they are. We started going to Nationwide in Columbus to see Dr. Jerry Mendell in 2017. At that point the gene therapies he was getting started on were the limb girdle sarcoglycanopathies, 2D,2E and 2C. The virus they use is able to fit those entire genes so in theory much easier than duchenne. There's quite a few TH-cam videos with Dr. Mendell describing the evolution of gene therapy. He is a saint.
His name definitely is talked about we have heard of him at conferences :) i believe it! we are really praying they can expand this to other MDs sooner than later! it is amazing what they are finally coming up with.
Its interesting to see that the behavior issues are common with DMD i did not know that. Im having alot of these struggles too!
It's so interesting to learn of the commonalities with DMD and other neurodevelopmental disorders like adhd! My guess is maybe due to the lack of dystrophin in the brain? The more we learn the more it starts to make sense!
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?
I'm sorry to hear that! These side effects are brutal. In our case it's pretty atypical with him only being 5 and confirmed to be caused by his prednisone so I'm not sure if exon skipping can be a factor 🤔