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    Adapting to Life Outside Our Home With SMA

    I once heard a joke that a woman’s mind is like an Internet browser that has 1000 tabs open and running at once. I laughed at the time, but came to realize that if I didn’t actually feel like this before hearing the short acronym “SMA,” I most certainly do now.

    I spend much of my time pre-planning and thinking of possible problems that we may run into because of the kids’ diagnoses. I think many other SMA parents do this as well. Prior to our lives with SMA, we didn’t realize just how inaccessible the world is to those who are differently-abled.

  • This topic has 22 replies, 8 voices, and was last updated 2 weeks, 1 day ago by Lupa F.

    • Author
      Posts
      • #21111
        Kevin Schaefer
        Keymaster

        Hey everyone!

        So I wanted to talk about Risdiplam, which is an SMA drug currently in the clinical trial phase. Developed by Roche and Genentech, this drug would be the first oral treatment for SMA, if FDA-approved. We hope to have more news about this drug in the coming months, but for now listen to this recent episode of the SMA News Today podcast, as we talk about it there.

        What questions do you have about Risdiplam? Is this treatment something you may look into for yourself, or for someone you know who has SMA?

      • #21115
        Rachel Markley
        Participant

        I don’t have a ton of questions – mostly cost, insurance approval, and drug interactions. And taste lol! Anything that avoids the words spine and needle in the same sentence and I’m down.

        • #21130
          Kevin Schaefer
          Keymaster

          I wonder about the taste too lol. I’m sure we’ll have more news about pricing and such in the coming months.

          By the way, how are your Spinraza treatments going? Have you experienced any changes lately?

          • #21138
            Rachel Markley
            Participant

            Kevin, I can move my left big toe! And my left arm contracture went from -40 to -20. Dose #5 is on the 13th!

          • #21139
            Kevin Schaefer
            Keymaster

            Rachel that’s great! Good luck on your next dose!

      • #21116
        Scott Inouye
        Participant

        My granddaughter is 3 yrs old type 2 and was diagnosed shortly after Spinraza was approved. She has had several treatments now and has seen significant gains. With new drugs being approved and studied, is there anybody looking at these in combination with Spinraza?

        • #21121
          DeAnn R
          Keymaster

          To be honest I’m not sure if there are studies looking at a Spinraza/Risdiplam combination. I’m doubtful though because from my understanding the two drugs have a similar function even though they do it differently. I have heard of a combo of Spinraza/Zolgensma but since Zolgensma is only approved for infants I haven’t been following results. Personally with a Spinraza/Risdiplam combo I would be concerned with excess protein causing kidney issues. It probably would be worth a study to see if there’d be a benefit though. I don’t have a link, but I recall hearing of a possible future combination therapy with Spinraza and Reldesemtiv. I can see combinations like that being more beneficial. If you do hear of something please share it with us!

      • #21134
        Kelly Miller
        Participant

        I would be so down with taking Risdiplam! I would say the major reason I haven’t pursued Spinraza is because of the injection issues. I had a spinal fusion at age 10 when they put an incredible amount of bone fragments from my shins along my spine, from end-to-end, billions of them from the very tip in my neck to the very bottom in my butt. So many doctors have tried to get epidurals & other injections for chronic pain into spots in my spine, and none have been successful. I just didn’t want to go thru the hassle. The other aspect was the side effects people have (& this is a big one too) from the injections. I deal with so much pain now just from sitting all these yrs. in the same position relatively that I can’t imagine having to deal with horrible headaches on top of it. It’s been easy so far to tell myself there just wouldn’t be that much of improvement in someone my age, keeping me from wanting it. Lately tho, with everything moving along for Zolgensma and now Risdiplam, it’s getting harder to put the idea out of my mind that maybe something would help me get rid of my pain or give me a little bit more strength & stamina when it comes to breathing and coughing… or both. I just so happened to get an email today from Biogen reminding people that we can get DNA testing done for free if we go thru them. And then, I see this post on here 🙂 I totally believe in messages coming from the great beyond, and I am truly feeling God prodding me into a different direction!

      • #21144
        Kelly Miller
        Participant

        So do y’all think the Risdiplam could be administered in feeding tubes? I was thinking about that this morning. I mean if it’s oral, you should be able to do it that way, right?

         

        • #21145
          DeAnn R
          Keymaster

          I think I read somewhere you could do it with a feeding tube, but of course I can’t find where I read it.

        • #21146
          Kevin Schaefer
          Keymaster

          Kelly, that’s a great question, and it’s not something I had thought about. If it’s oral, I don’t see why it couldn’t be delivered via feeding tube. But it’s something I’ll look into. Thanks for bringing this up!

      • #21147
        Kelly Miller
        Participant

        Thanks Kevin, but I found my answer. It actually states on CureSMA’s website, in a section about Risdiplam, that it can be administered thru a feeding tube!
        I started doing a little research after I wrote my question. Didn’t want you to look unnecessarily.

      • #21444
        Olga
        Participant

        Dear All,

        I have a brother with SMA 2 who can’t get Spinraza because of his spinal deformity (he had spinal surgery and our doctors can’t give him medicine because of it). We are looking for another way to stop this terrible disease. Please advise what should we do? Where can we go to get help? Has anyone had such a situation and how did you deal with it? I hope Ridisplam will be on our market as soon as possible because it is probably the last option to help my brother.

        • #21445
          DeAnn R
          Keymaster

          Olga, sorry to hear your brother is unable to get Spinraza.  Have the doctors considered alternative administration methods?  I’ve had my spine fused and get the injection through my side called the transforaminal approach.  Others have had a port placed.  Some get cervical injections.  Sometimes a CT scan can locate an opening as well.  You’re not alone though.  I know others who can’t get Spinraza either.  Hugs!

          If indeed it’s not possible I do have some good news regarding risdiplam.  I heard it’s gotten okayed for fast track FDA approval.  I know for many it can’t come fast enough, but it shouldn’t be too far off now.

          • #21446
            Olga
            Participant

            We tried to create a port, but the operation to create it was too risky. We will continue to look for some other method. Thank you for your support, I hope you are right that oral treatment – Risdiplam, will be introduced soon.

          • #21452
            Alyssa Silva
            Keymaster

            Hi Olga. While there isn’t another drug like Spinraza on the market, has your brother tried albuterol syrup? I haven’t had enough firsthand experience with it to vouch for its effectiveness, but I know many people with SMA use it and experience a slight increase in their stamina. Perhaps a little boost in energy could help him until Risdiplam becomes available?

          • #21490
            Olga
            Participant

            Dear Alyssa Silva and Kelly Miller,

            Thanks for the advice, I haven’t heard anything about albuterol syrup. This is good news that it can help! We will ask our doctor about this syrup. I hope that soon there will be a solution and that my brother and others will start treatment asap.

             

             

          • #21494
            Alyssa Silva
            Keymaster

            Best of luck to your brother, Olga! I’m happy to help whenever I can. 🙂

      • #21462
        Kelly Miller
        Participant

        Olga,

        I’m sorry to hear your brother can’t have the Spinraza injections as he would like. I’ve also had a spinal fusion that put lots of pieces of bone in my back, along my spine to make it grow straighter. Because of this, any kind of injection in my back is next to impossible. Drs. have tried before to do epidurals and cortisone injections with no success whatsoever. Since it has never worked in the past, I made the decision to sit out the Spinraza treatment myself and wait for Risdiplam to be approved. I’m really happy to hear that it’s been fast-tracked, and I’m sure that will be great news for your brother as well.

        I CAN speak from personal experience about the albuterol syrup Alyssa mentioned. I’ve been taking it now for about a little over a year, and I definitely can tell a difference in my stamina. In fact, I quit taking it for about 3 mos. when we moved from Texas to Florida – I ran out, I didn’t have a new pulmonologist yet, other things were more important at the time. When I finally got an appt with a new doctor, my husband told him that he noticed a big change in me when I didn’t take the syrup. He said I have more energy and breathe a lot better when I’m taking it! Neither 1 of my pulmonologists (not Texas nor Florida) had ever heard of the medicine having that effect on someone, but both were willing to give it to me with the idea that it had helped other people with SMA (as per my info from social media). This might be something your brother could really benefit from if he can get a prescription. Talk it over with his doctor and see what would be best. It’s worth a try until he can get Risdiplam.

      • #21460
        Lupa F
        Participant

        Just want to note since I can’t find a good place to contact someone about it, the new article about Risdiplam has a major error about Spinraza: ”

        “Spinraza works much the same way, and is given as an intrathecal injection into the spinal cord. Treatment is once a month after an initial loading dose.”

        They even link to the Spinraza prescribing information and it says clearly on the 1st page: “Initiate SPINRAZA treatment with 4 loading doses; the first three loading doses should be administered at 14-day intervals; the 4th loading dose should be administered 30 days after the 3rd dose; a maintenance dose should be administered once every 4 months thereafter”.

        • #21472
          Kevin Schaefer
          Keymaster

          Hey Lupa, thanks for the heads up. Our news team is fixing this.

          • #21507
            Lupa F
            Participant

            Hey Kevin,

            Thanks, I just want people to have accurate information since it might affect people’s decisions on treatments. I checked and they did update it, but it’s still wrong. It now says: “Sprinraza works much the same way, and is given as an intrathecal injection into the spinal cord. Treatment is once every four months after an initial loading dose.” As all of us that have gotten it know, it’s not a single loading dose which is what this implies. All they need to say is “after 4 initial loading doses.”

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