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The importance of newborn screening for SMA

Rory and Carolyn met in seminary, fell in love, and got married. They decided to hold off on starting their own family while they settled into their first jobs in North Dakota. After a few years, they moved to Minnesota, where Rory accepted a job as a minister, and Carolyn enrolled in a hospital chaplain residency program. At the time, they did not know how important the decision to move would be for their future family. When they moved, neither North Dakota nor Minnesota had SMA on their newborn screening panel. It wasn’t until March 2018 that Minnesota adopted and implemented newborn screening for SMA, and then several months later, in 2018, Rory and Carolyn’s only son Edan was born.


This topic has 7 replies, 6 voices, and was last updated 1 month, 2 weeks ago by DeAnn R.

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    • #24500
      DeAnn R
      Keymaster

      Since Evrysdi (risdiplam) is FDA approved, many are considering the switch from Spinraza. Myself included. However, Spinraza works well for me.
      Even though the process is a pain, I don’t have ill effects or some of the other common issues. Therefore I’ve decided to bide my time and take in all of the considerations before jumping ship. My regular neurology appointment should be in December, so I think I’ll discuss it then.
      As a parent I can’t imagine what a difficult decision this would be. There are so many factors to consider. Obviously a spinal injection isn’t optimal. If Spinraza is working well though switching might not appeal to you. Then there’s the side effects to think about. I haven’t dived into researching yet, but it’s always something to look into.
      Have you made the decision? What factors influenced your choice?

    • #24513
      Dennis Turner
      Participant

      My Neurologist suggested waiting a year to see other people’s results.

      For me waiting isn’t a huge sacrifice, Spinraza seems to be working quite well and like you, I am tolerating treatment at this time.

    • #24514
      Mike Huddleston
      Participant

      Hey DeAnn – Thanks for posting this as it’s a critically important subject. Oh, if it were 5 years ago when we weren’t even sure treatment options would be available. 🙂

      Like you, I am doing well on Spinraza with little if any side effects and I (so far) have handled the injections well.  Including the initial loading doses, I’ve had a total of 10 so far.  And the results for me, as I’ve indicated in other posts here, have been pretty remarkable.  I also have never dealt with the reduction in energy when approaching that final period before the next treatment, so I’ve had stability in that regard as well.  That said, there is some appeal in considering a switch to Evrysdi. Daily at home is just one of them (or is that two??).

      I’ve briefly discussed this with both Dr. Tom Crawford, who runs the Spinraza program out of Johns Hopkins in Baltimore and my regular neurologist, Dr. Charlotte Sumner, also at Hopkins.  The concern, if it can be categorized as such, by both of them is the longer term effect of Evrysdi. There is basically just more experience over a longer period of time with a larger patient population for Spinraza.  That carries some weight, especially for those having success and tolerating Spinraza well.

      I will be discussion in more detail next time I see  Dr. Sumner at my annual clinic visit this fall.  In the meantime, I have no immediate plans to switch, but like you, will sponge up as much additional info as possible as it becomes available.

      Edit: I forgot to mention that my main improvements have been in my lower body, with some slight weakness still progressing primarily in my shoulders.  I will also be adhering to my next maintenance dose which will be in September.  Other factors will also be the results of current studies (like increase dose amounts) for Spinraza to determine potential increased efficacy.

    • #24516
      Dick
      Participant

      Like many of you who have already posted, I have not had any difficulty taking my Spinraza shots nor any unpleasant after effects. The hospital where I have been receiving those treatments is 40 minutes from my home, so travel has not been a difficulty. However, in these days of a pandemic, I would much rather take a drug I can administer at home then have a visit to a hospital, even if it were walking distance from my home. This is one factor in considering the switch. And, unlike all of you who have already posted, I have had all four loading doses and four subsequent maintenance doses,  but Spinraza does not seem to be helping me in any noticeable way. I have type III which has been very slowly progressive throughout my life. (I am 81 years old, and was the oldest person in the world taking Spinraza treatments), and I have at this age lost so much, or in my words so many of my muscles are already dead, that Spinraza does not seem to be able, at least at current dosage levels and frequency, to revive any capability of which I am aware. Even before Evrysdi was approved, I had decided, and my neurologist said the decision was reasonable, to cease Spinraza treatments and wait for FDA approval of Evrysdi. At 81 I’m not terribly concerned with unknown long-term effects. So now I must wait for Medicare approval of payment for this new treatment, and then find a pharmacy authorized to prepare the compound that will accept a Medicare patient, which means they must pay for the drug before being reimbursed.

    • #24517
      Alyssa Silva
      Keymaster

      DeAnn. I am in the exact same boat as you are. In fact, my upcoming column on Friday is all about why I’m sticking to Spinraza for now. I was actually just reading the Evrysdi package insert, and I gotta be honest… the side effects made me raise an eyebrow. If I’m doing well on Spinraza, do I risk it all for something that isn’t guaranteed?

    • #24518
      Lupa F
      Participant

      Alyssa, I’m sure if you looked at the list of side effects for a lumbar puncture or Spinraza there would be a huge list of stuff too. They have to list any side effects anybody had, even if almost nobody gets them. That’s why the commercials for drugs are 2 seconds of describing it, then 28 seconds listing side effects.

      One thing I thought of was that Spinraza is covered under Medicare Part B because it’s part of a medical procedure. Evrysdi I think would be under Part D. Not sure if that’s going to cause any problems.

      Dick, Evrysdi is being processed by a specialty pharmacy, it’s mentioned in the articles. We can’t just go up to a CVS and request this. So I think they’ll know how to deal with Medicare.

      • This reply was modified 1 month, 2 weeks ago by Lupa F.
    • #24520
      Alyssa Silva
      Keymaster

      Oh yeah. I looked at Spinraza’s side effects as well. I know they have to list every possible scenario, I just hadn’t been aware of Evrysdi’s yet.

    • #24521
      DeAnn R
      Keymaster

      Lupa, I was wondering the same thing about Medicare coverage with Spinraza being covered under part B. Guessing not all drug plans will cover it. Also wondering on copays. I’m not all that familiar with the drug coverage portion of Medicare.

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