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.
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i saw this on your facebook page. fantastic news!
Summary from sciencemag.org.
Hope it is a difference-maker.
SMA has just made an appearance on PostSecret.
I’m very excited about the Spiranza news. I passed it on to a family member who has a child with SMA.
Excellent! Getty’s docs are working on getting it.
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$750k per year to treat
Yikes!
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Hopefully it’s not too late for her.
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.
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.
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Happy Birthday, Getty! This is double good news. Please keep us posted.
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.
How exciting! Happy birthday, Getty! I thought of you when I read about this treatment. Good luck, to the Family Bearflag!
Happy Birthday Miss Getty! May your day be just wonderful!
I shall dance in my kitchen in your honour. :):):):):):)
Good news is always welcome! Congratulations all around!!
What a wonderful thing. Go Getty!
Terrific news.
So assuming that this treatment works, what is Getty’s prognosis?
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!
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!
Bearflag - How long before you should start to see improvements?
StG