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  • UK Denies Spinraza as SMA Treatment

    Posted by deann-r on August 16, 2018 at 9:17 pm

    One step forward and two steps back.  Even though I can’t take a step, that’s what the recent news feels like that NICE (National Institute for Health and Care Excellence) does not recommend Spinraza to be funded by NHS (National Health Services) in England and Whales.  After reading some of the supporting documentation it really boils down to cost effectiveness.  Apparently the committee hasn’t had a loved one struggling to breathe while nurses and doctors rush in to clear their airway.  Or they haven’t been positioned  uncomfortably in bed while just moving an inch would relieve pressure so it doesn’t feel like your tailbone is about to pop out of your skin.  They haven’t had to come to terms the fact while other kids are playing kickball they need to have a nurse assist them with a bowel movement or breathing treatment.

    When I hear the words cost effective I think of something like a cell phone, not human life. Believe me, I’ve grappled with the expense of this treatment.  I’m then reminded of my little brother who passed away from SMA complications when he was just 4 years old.  My parents had to watch him struggle to take his last breath.  Back then there was no treatment for SMA.  Now there is.  No parent should have to bury their child without being able to give them every opportunity to thrive.  By saying this treatment isn’t cost effective, they’re taking away the only current option to halt or even reverse some of the effects of this disease.

    For those who don’t have access to Spinraza, I’m a testament that you can live a fulfilled life.  Will it be easy?  No, but it is possible.  Don’t give up.  At 38 years old (type 2) I received my first dose of Spinraza.  I’m not doing jumping jacks like NICE apparently wants to see.  However, almost a year after starting the treatment I came down with a severe cold.  Pre-Spinraza a cold like this would have landed me in the ER.  Not this time.  Anyone who has SMA knows little things like that mean the world.  NICE can shove cost effective up their you know what as far as I’m concerned.  Now that I’m on Spinraza maybe I could do it for them.  I have increased 8 points on my upper limb scale.

    While NICE wants to see more evidence of the long term effectiveness of Spinraza children are dying.  SMA is the leading genetic cause of infant death and something can finally be done about it.  In episode 6 of the Morale Monologue Michael discusses this decision by NICE.  Although it’s a major setback it’s not a decision set in stone.  Watch his video, and use these links to help bring Spinraza to our UK family.

    SMA Trust website:

    National Institute for Health and Care Excellence website:


    kevin-schaefer replied 5 years, 8 months ago 2 Members · 1 Reply
  • 1 Reply
  • kevin-schaefer

    August 17, 2018 at 1:10 pm

    This news was truly disheartening. It’s astounding that people could look at this from a cost-effective standpoint. We can only hope that NICE will reverse this decision. Thanks for posting.

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