# My baby girl has Spinal Muscular Atrophy (SMA) **URL:** \ **Category:** Miscellaneous and Personal Stuff I Must Share\ **Created:** [July 18, 2010, 3:17pm UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126 "2010-07-18T15:17:44Z")\ **Posts on this page:** 20\ **Page:** 51
**Author:** ![Bearflag70](https://avatars.discourse-cdn.com/v4/letter/b/8e7dd6/32.png) [@Bearflag70](https://boards.straightdope.com/u/Bearflag70)\ **Post date:** [December 24, 2016, 1:52am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1001 "2016-12-24T01:52:20Z")
I was the lifeline. I Flew to Vegas overnight for filming. In other news, FDA just approved the first ever SMA TREATMENT!! It’s called SPIRANZA. Sent from my iPhone using Tapatalk ---
**Author:** ![rocking\_chair](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/rocking_chair/32/2918_2.png) [@rocking\_chair](https://boards.straightdope.com/u/rocking_chair)\ **Post date:** [December 24, 2016, 2:18am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1002 "2016-12-24T02:18:50Z")
i saw this on your facebook page. fantastic news! ---
**Author:** ![Baal\_Houtham](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/baal_houtham/32/13604_2.png) [@Baal\_Houtham](https://boards.straightdope.com/u/Baal_Houtham)\ **Post date:** [December 24, 2016, 4:42am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1003 "2016-12-24T04:42:19Z")
> [@Bearflag70](#): > > I was the lifeline. I Flew to Vegas overnight for filming. > > In other news, FDA just approved the first ever SMA TREATMENT!! > > It’s called SPIRANZA. Summary from [sciencemag.org](http://www.sciencemag.org/news/2016/12/novel-drug-rescues-babies-fatal-neurodegenerative-disease). Hope it is a difference-maker. ---
**Author:** ![Quartz](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/quartz/32/267_2.png) [@Quartz](https://boards.straightdope.com/u/Quartz)\ **Post date:** [December 26, 2016, 9:12am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1004 "2016-12-26T09:12:35Z")
SMA has just made an appearance on [PostSecret](http://postsecret.com/2016/12/24/classic-secrets-156/www-petitiontocuresma-com/#main). ---
**Author:** ![carnut](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/carnut/32/3531_2.png) [@carnut](https://boards.straightdope.com/u/carnut)\ **Post date:** [December 26, 2016, 10:41pm UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1005 "2016-12-26T22:41:31Z")
I’m very excited about the Spiranza news. I passed it on to a family member who has a child with SMA. ---
**Author:** ![Bearflag70](https://avatars.discourse-cdn.com/v4/letter/b/8e7dd6/32.png) [@Bearflag70](https://boards.straightdope.com/u/Bearflag70)\ **Post date:** [December 28, 2016, 5:29am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1006 "2016-12-28T05:29:31Z")
Excellent! Getty’s docs are working on getting it. Sent from my iPhone using Tapatalk ---
**Author:** ![Bearflag70](https://avatars.discourse-cdn.com/v4/letter/b/8e7dd6/32.png) [@Bearflag70](https://boards.straightdope.com/u/Bearflag70)\ **Post date:** [December 29, 2016, 6:22am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1007 "2016-12-29T06:22:39Z")
$750k per year to treat Yikes! > **[Informa Connect - Know more, do more, be more.](https://informaconnect.com)** > > Informa Connect is a live events, digital content specialist connecting professionals with knowledge, ideas and opportunities. Sent from my iPhone using Tapatalk ---
**Author:** ![Quartz](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/quartz/32/267_2.png) [@Quartz](https://boards.straightdope.com/u/Quartz)\ **Post date:** [December 29, 2016, 10:57am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1008 "2016-12-29T10:57:25Z")
Hopefully it’s not too late for her. > [@](#): > > It’s important to note that a drug’s list price isn’t the actual price once insurers and their agents negotiate discount deals and rebates. But those negotiations typically remain secret, so real-world drug prices are rarely publicized. ---
**Author:** ![Iggy](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/iggy/32/3364_2.png) [@Iggy](https://boards.straightdope.com/u/Iggy)\ **Post date:** [December 29, 2016, 12:47pm UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1009 "2016-12-29T12:47:18Z")
For what little it matters, the actual production cost is relatively minimal. It’s all the R&D, drug company profit, and the fact that it is a relatively small market that is driving the pricing. Spiranza is a specific short RNA sequence, an antisense oligionucleotide, and could be mass produced much easier and cheaper than some of the newer bio-engineered medications that have hit the market such as the antibody drug conjugates used in certain cancer treatments. Hopefully this means that Biogen will have a lot of wiggle room in pricing and patient assistance programs. ---
**Author:** ![Bearflag70](https://avatars.discourse-cdn.com/v4/letter/b/8e7dd6/32.png) [@Bearflag70](https://boards.straightdope.com/u/Bearflag70)\ **Post date:** [March 26, 2017, 1:38am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1010 "2017-03-26T01:38:04Z")
For the record, Getty turns 7 today and got her 1st Spinraza treatment 4 days ago. Thanks for sticking with us and offering encouragement the past 7 years. Sent from my iPhone using Tapatalk ---
**Author:** ![Mighty\_Girl](https://avatars.discourse-cdn.com/v4/letter/m/7bcc69/32.png) [@Mighty\_Girl](https://boards.straightdope.com/u/Mighty_Girl)\ **Post date:** [March 26, 2017, 2:25am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1011 "2017-03-26T02:25:57Z")
Happy Birthday, Getty! This is double good news. Please keep us posted. ---
**Author:** ![lavenderviolet](https://avatars.discourse-cdn.com/v4/letter/l/cdc98d/32.png) [@lavenderviolet](https://boards.straightdope.com/u/lavenderviolet)\ **Post date:** [March 26, 2017, 2:26am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1012 "2017-03-26T02:26:55Z")
> [@Bearflag70](#): > > For the record, Getty turns 7 today and got her 1st Spinraza treatment 4 days ago. Thanks for sticking with us and offering encouragement the past 7 years. I remember reading this thread when it all started years ago. I’m so glad that there is finally a treatment available for this condition. All the best to you and your family. ---
**Author:** ![Merneith](https://avatars.discourse-cdn.com/v4/letter/m/57b2e6/32.png) [@Merneith](https://boards.straightdope.com/u/Merneith)\ **Post date:** [March 26, 2017, 2:45am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1013 "2017-03-26T02:45:24Z")
How exciting! Happy birthday, Getty! I thought of you when I read about this treatment. Good luck, to the Family Bearflag! ---
**Author:** ![kambuckta](https://avatars.discourse-cdn.com/v4/letter/k/53a042/32.png) [@kambuckta](https://boards.straightdope.com/u/kambuckta)\ **Post date:** [March 26, 2017, 2:51am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1014 "2017-03-26T02:51:01Z")
Happy Birthday Miss Getty! May your day be just wonderful! I shall dance in my kitchen in your honour. :):):):):):)🙂 ---
**Author:** ![Typo\_Knig](https://avatars.discourse-cdn.com/v4/letter/t/cdc98d/32.png) [@Typo\_Knig](https://boards.straightdope.com/u/Typo_Knig)\ **Post date:** [March 26, 2017, 8:53pm UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1015 "2017-03-26T20:53:30Z")
> [@Bearflag70](#): > > For the record, Getty turns 7 today and got her 1st Spinraza treatment 4 days ago. Thanks for sticking with us and offering encouragement the past 7 years. > Sent from my iPhone using Tapatalk Good news is always welcome! Congratulations all around!! ---
**Author:** ![kayT](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/kayt/32/3633_2.png) [@kayT](https://boards.straightdope.com/u/kayT)\ **Post date:** [March 26, 2017, 8:57pm UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1016 "2017-03-26T20:57:38Z")
What a wonderful thing. Go Getty! ---
**Author:** ![Lochdale](https://avatars.discourse-cdn.com/v4/letter/l/edb3f5/32.png) [@Lochdale](https://boards.straightdope.com/u/Lochdale)\ **Post date:** [March 28, 2017, 5:13pm UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1017 "2017-03-28T17:13:45Z")
> [@Bearflag70](#): > > For the record, Getty turns 7 today and got her 1st Spinraza treatment 4 days ago. Thanks for sticking with us and offering encouragement the past 7 years. > Sent from my iPhone using Tapatalk Terrific news. So assuming that this treatment works, what is Getty’s prognosis? ---
**Author:** ![Infovore](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/infovore/32/420_2.png) [@Infovore](https://boards.straightdope.com/u/Infovore)\ **Post date:** [March 28, 2017, 5:17pm UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1018 "2017-03-28T17:17:08Z")
Wow. I’ve been following your story since you first posted about it here. I just looked back to the beginning of this thread, where you were talking about a prognosis of 2 years. And now here she is 7! That is great news. Happy birthday, Getty! ---
**Author:** ![carnut](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/carnut/32/3531_2.png) [@carnut](https://boards.straightdope.com/u/carnut)\ **Post date:** [March 30, 2017, 3:20am UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1019 "2017-03-30T03:20:32Z")
Oh wow, what a great birthday present (I loved the pic of the mermaid cake too). Happy birthday Getty. I hope you got mermaid gifts! ---
**Author:** ![StGermain](https://sea3.discourse-cdn.com/straightdope/user_avatar/boards.straightdope.com/stgermain/32/2868_2.png) [@StGermain](https://boards.straightdope.com/u/StGermain)\ **Post date:** [March 30, 2017, 2:24pm UTC](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126/1020 "2017-03-30T14:24:27Z")
**Bearflag** - How long before you should start to see improvements? StG [Previous page](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126.md?page=50) [Next page](https://boards.straightdope.com/t/my-baby-girl-has-spinal-muscular-atrophy-sma/547126.md?page=52)