SMA News Forums Forums Treatments and Research Spinraza (nusinersen) Effects on Adults with SMA

  • Effects on Adults with SMA

    Posted by kevin-schaefer on February 12, 2018 at 2:02 pm

    Hello all. I wanted to address other SMA adults who are receiving Spinraza. I know there are a lot of articles and discussions out there about kids who receive this treatment, but I’d like to hear from other adults.

    I’m 24 and started my Spinraza treatments in November 2017. Thus I’ve completed my loading doses, and my fifth dose is scheduled for May. Since starting I haven’t noticed any major changes in my physical strength, but I have experienced a general boost in energy. This is pretty significant for me, and I’d love to hear stories from other users here. Also feel free to ask questions here if you’re still trying to acquire the treatment.

    deann-r replied 5 years, 11 months ago 6 Members · 22 Replies
  • 22 Replies
  • pooja-arora

    Member
    February 14, 2018 at 1:26 am

    I am SMA Type 3 or Type 4 sufferer.

    I am looking for treatments. presently I  do all my chores myself.

    • kevin-schaefer

      Member
      February 14, 2018 at 2:27 pm

      Thanks for joining the discussion Pooja!

      Yes, just to echo what DeAnn said above, Spinraza is a great treatment which I started receiving toward the end of 2017. I’m also happy to answer any questions you have about it. Welcome to this page.

    • pooja-arora

      Member
      February 22, 2018 at 2:30 am

      Kevin, what improvement you feel.

      I mean if you could tell in details that what actions/ movements which you were facing difficulties earlier and is improved after Spinraza treatment.

      • kevin-schaefer

        Member
        February 22, 2018 at 10:13 am

        Hi Pooja. In regards to your question about what improvements I’ve seen since starting the treatment, I’d say the biggest is an increase in my energy levels. I don’t feel nearly as tired at the end of the day as I used to, even when I’m out all day.

        Another thing my friends and family have noticed is that my speech has improved. Because I’m not running out of breath, my speech isn’t slured like it used to be. I haven’t seen any major changes related to my arm strength or physical abilities, but these other changes have certainly helped. Thanks for asking! Feel free to let me know if you have any other questions.

  • deann-r

    Member
    February 14, 2018 at 10:09 am

    Great to hear from you Pooja Arora!  Currently Spinraza is the only FDA approved treatment for SMA.  Although other treatments are in trials they have not yet been FDA approved.  Spinraza has been approved for all ages and types (with rare exclusions) of SMA, however access really depends on what country you live and what insurance you have.  Some are putting age and type restrictions on who can receive treatment.  From my experience in adults improvements with Spinraza are a bit slower coming than what children are seeing.  However, since SMA is progressive, slowing or stopping progression is a big deal.  That’s what Spinraza is aiming to do.  If you have further questions please ask!

  • pooja-arora

    Member
    February 22, 2018 at 2:28 am

    Hi I am from India, and adult with Type 3 SMA.

    Please let me know if I can also go for Spinraza clinical trial.

  • deann-r

    Member
    February 22, 2018 at 10:20 am

    Pooja, currently there are no ongoing trials for Spinraza as it is already approved.  Your access to it really depends on the healthcare system in your country.  Unfortunately many countries are limiting access to those who are type 1 infants.  I would recommend contacting your doctor to start the discussion.  In adults improvements have been slower, but still show promising results.

    Personally I have only seen minor improvements in arm function.  Progression has seemed to stop though.  It’s difficult knowing there’s a treatment out there you haven’t been able to access, but continue holding on to hope.

    Thanks for reaching out.  Please don’t hesitate to ask more questions.

  • dennis-turner

    Member
    March 22, 2018 at 9:13 am

    I am also following this topic.

    I was diagnosed with SMA3  in 1975 and was able to walk much of the time until  about 5 years ago. I am now 59 years old.

    My Dr. is talking with me regarding spinraza.

    Maybe/hopefully I will get treatments once approved by both him and my insurance.

    • deann-r

      Member
      March 22, 2018 at 10:47 am

      Dennis, I think it’s great that you’re checking into Spinraza.  For me it’s been like hitting the pause button where SMA is concerned. Over time I think gains will come.  Have you met with a neurologist? If you were diagnosed back in the 70’s they may require an updated genetic test like I had to do. Easier to get insurance approval .

    • kevin-schaefer

      Member
      March 22, 2018 at 12:56 pm

      Thanks for commenting Dennis. And yeah echoing what DeAnn said, meeting with a neurologist would be good. My neurologist has been really helpful throughout the Spinraza process.

      Keep us updated. We’re happy to answer any questions you have.

  • dennis-turner

    Member
    March 22, 2018 at 7:10 pm

    I meet with my Neurologist Tuesday. Thank you all for your encouragement.

    • deann-r

      Member
      March 22, 2018 at 8:14 pm

      Keep us updated, and best of luck on your journey. Don’t hesitate to ask any questions!

  • valeriy-evdokimov

    Member
    March 27, 2018 at 9:38 am

    Unfortunately in Russia this treatment is not available((

    • deann-r

      Member
      March 27, 2018 at 10:05 am

      I’m hoping over time it will be more readily available around the world.

  • dan-dan

    Member
    March 27, 2018 at 1:12 pm

    Hi DeAnn, since your last apdate about your situation is from last month, can you tell us if you can see any improvments yet? I am a type 3 who is interested to try spinraza.

    TNX

    • deann-r

      Member
      March 27, 2018 at 2:33 pm

      Thanks for asking Dan.  As far as obvious improvements I haven’t seen anything really significant.  Sometimes when I pick something up it seems lighter than I expect it to.  Friends have mentioned I move my “bad” arm more and sound a little louder.  Even though my pulmonary function test didn’t show improvement I feel my cough has more force to it.  So, subtle things.  My calves have been cramping a bit too.  Nothing ultra painful, but noticeable.  I take it as them waking up.

      I encourage you to look into Spinraza.  From what I’ve heard many type 3 individuals have experienced increased endurance and energy.  Someone described it like sandbags being lifted off their legs.  Seems results vary from person to person, but positive from what I’m seeing.

      Feel free to ask any questions.  My next vlog will detail the process I go through.

       

  • dennis-turner

    Member
    April 10, 2018 at 9:52 pm

    Update from my Neurologist.  Apparently there are no adult trials going on in the Boston area. He is currently working to start the process.

    • deann-r

      Member
      April 11, 2018 at 8:24 am

      Dennis, glad to hear you’ve inquired about Spinraza.  Good news, Spinraza is actually approved for adults already, so no trial necessary.  If your doc is willing, he should be able to become an administration site.  I would suggest maybe having them contact Biogen, the pharmaceutical company.  Also, here is a link to sites that are administering Spinraza http://www.curesma.org/spinraza/sites/  It helped me out when I started my journey.  Best of luck, and keep us posted!  Please ask if you have questions along the way.

  • dan-dan

    Member
    April 12, 2018 at 1:37 pm

    DeAnn, did you notice any increase of protein in your spine fluid and/or in your Urine?

    • deann-r

      Member
      April 12, 2018 at 4:48 pm

      Dan, My last urine test did show the protein level had increased slightly, but nothing significant.  The doctor didn’t even mention it, I was just comparing lab results.  As far as spinal fluid, I don’t get results from that, so couldn’t tell you.  In my mind that tells me Spinraza is doing what it’s supposed to.  Fingers crossed anyway.  Thanks for asking, and don’t hesitate asking more questions!

  • pooja-arora

    Member
    April 18, 2018 at 12:43 am

    How is treatment charges are managed, as it is very very high and also not available in Inida?

    • deann-r

      Member
      April 18, 2018 at 7:31 am

      Pooja, in the United States most individuals need to get approval through their insurance companies. In some instances if it’s been denied the pharmaceutical company has stepped in.  In other countries where the treatment has been approved it’s really based on how their health system works.  Some have put many restrictions on who can get treatment.  I’m sorry to hear it’s not yet available to you.  Keep advocating for it, and as new treatments arise hopefully it will become more readily available. Thanks for asking and best wishes on your journey.

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