Good luck with the treatment and Happy Birthday!
Hope is a great thing. Wishing your family a wonder filled 2017.
Unknown. One of the biggest factors in treating SMA is early intervention. Get the treatment going before motor neurons start failing and dying off.
Getty is an “older type 1” and for treatment impacts, we will just have to watch and hope for the best.
The hope is that her existing motor neurons will get a boost, allowing for a better signal and more muscle control and strength. To the extent some motor neurons are not quite dead, we hope they can be salvaged, which could result in some gains. For motor neurons that have permanently failed due to her age, we expect nothing.
Realistically, we hope for two things: (1) halting progression of weakness; and (2) some additional head, jaw, mouth, hand, wrist, respiratory, and foot strength, and some additional leg and arm movement and endurance and postural strength would be awesome.
We are excited for her opportunity but don’t want to get too caught up in wishful thinking. Simply halting progression would be great!
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We think we have seen minor improvements after one dose. With her hand laying flat on her belly, she can usually lift her index and middle fingers. After the first dose, she was getting some wrist lift. Also, her jaw seems to be more mobile; she was wiggling it around more than usual.
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Those apparent gains are subtle nuances we can see because we know her so well. On the other hand, we realize we could be biased by wishful thinking and we are recording things we think could be improvements.
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I should probably document her abilities over time with various isolated joint movements under controlled conditions.
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Thinking good thoughts that you are seeing real improvements. Documenting what you see is a great idea. Maybe write down what you know now about her abilities, then keep track on a regular schedule, so you’ll have something to measure her progress.
Best wishes to you all.
I think documenting is a great idea, particularly if you may have to fight with your insurance to continue treatment. If you show positive gains, they’re more likely to approve it.
StG
Word is now that they will establish baselines and monitor results to justify continued use.
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Yes, indeed. I’m sure the results will add immeasurably to our understanding of this potential treatment.
Happy Birthday Getty Owl!!
A joyous 8th Birthday to you, precious one! So happy to celebrate with you today!!
The is the best news ever!! I’ve got to go full Roger Daltrey - YouTube
HAPPY BIRTHDAY GETTY OWL!!!
I’m always glad to see happy milestones in this thread.
Happy Birthday Getty!!
HAPPY BIRTHDAY MISS GETTY. Hope you have a wonderful day. Fancy you being 8 already!!
WOOOO HOOOO :):):):)
Thank you all! It’s been quite a ride. Our lives are totally different now than 2010. She’s a happy kid and that’s what matters most.
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How’s the Spinraza doing for her?
StG
Good, thanks!
She seems to have recovered a bit more strength in hands, feet, and hips… not a full point on the CHOP INTEND scale, but we weren’t expecting that kind of progress.
Preventing further decline is progress.
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Yes, yes, that IS what matters most. Good to hear that the treatment is having favorable result, here’s to the Owl and to her folks!
Good heavens, 8 years already! Happy birthday!