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Pediatric neurodegenerative disorders


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When we were at NIH for a study, we were on the same treatment cycle as a number of families who were part of NIH's rare diseases research. A lot of those patients were kids with ped neurodegenerative diseases, many of which are still nameless.  There is an entire research division for such things, and a number of really good resources.  In talking with those families all of them talked about how hard it was to get accurate information and how they wished they had known earlier about treatment centers and aid for medical transport.

 

Here's a link to NIH's page for such things: https://rarediseases.info.nih.gov/resources/5/support-for-patients-and-families#category16

 

ETA: I'm sorry you're going through this cr*p.  I wish I could bring you guys dinner and some good chocolate.

Edited by kbeal
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Friends' dc has a neurodegenerative disorder - I can't remember exactly which one it is, but she's one of only a few in the country - and finally finding someone who could give real answers made a huge difference for them.  I pray you are able to find the right answers for your dc.

 

 

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There is quite a spectrum of diagnoses that fall under this - some are much better and more livable and some are unspeakably horrible. Praying for you and your sweet children :grouphug:

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Please don't quote-

 

My ds was tested for several possible ones, including the one you mentioned. (I don't want to quote.) They involved going to a geneticist and all three of us- me, dh, and ds, giving blood samples.  There were also lots of other possibilities and we were in many offices and hospitals.  It turned out to be a seizure disorder that he has outgrown.  

 

It is terrifying, and I'm so sorry that you have to deal with this.  It is so serious that every physician gave us home and cell numbers.  We had laminated cards printed from doctors that enabled us to go straight to the hospital lab for testing if he was displaying certain symptoms.  Even if your background is medical, you will learn more than you want to know and will realize how little most doctors know, because they don't have to deal with it very often.  We had a fabulous neurologist who was determined to find the cause of his issues.  I will warn you- a couple of times we'd get a call to bring him in because he had an idea. Then he would be admitted for testing. 

 

Keep the faith- whatever that means for you! Don't lose hope!  I hope it turns out to be nothing!

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We won't really know anything for a while.

I have a friend with two boys with a leukodystrophy and she is actually the one who first saw some red flags and started pushing me to see if there is more than just high functioning autism going on. Especially since DS5 doesn't quite fit with HFA; his issues are mostly behavioral and it is getting worse. Plus headaches, weight loss, sudden loss of coordination and fine motor skills. We've ruled out brain tumor. We have another appt on Wednesday; I'm trying to gather information before then.

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:grouphug: My son has a metabolic neuromuscular condition that is degenerative. We had really scary possibilities presented as likely explanations when he had his initial testing (Cleveland Clinic). It was terrifying.The local children's hospital admitted shortly thereafter and repeated those labs plus some. The horrible things they initially suspected were ruled out in skin bx. The end result was still pretty grim prognosis wise, but his course has been much better than we were told to expect. The geneticist is more hopeful and positive every single appointment.

 

My point is empathy. The possibilities and uncertainties that you are in now were the hardest part for me. I really hope it's something different than what they suspect, as it was for us. :grouphug:

Edited by sbgrace
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We won't really know anything for a while.

I have a friend with two boys with a leukodystrophy and she is actually the one who first saw some red flags and started pushing me to see if there is more than just high functioning autism going on. Especially since DS5 doesn't quite fit with HFA; his issues are mostly behavioral and it is getting worse. Plus headaches, weight loss, sudden loss of coordination and fine motor skills. We've ruled out brain tumor. We have another appt on Wednesday; I'm trying to gather information before then.

We will be praying for you on Wednesday. God bless your friend for mentioning it, because catching these as soon as possible helps with treatment and he is still just a little guy. But I'm sincerely hoping she was wrong and they'll find nothing of consequence wrong with him :grouphug:

 

Your sweet family has been through the wringer and it just stinks. You deserve good news :(

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