March 12, 2020 at 7:00 am #22654DeAnn RKeymaster
As you may be aware Roche/Genentech’s treatment risdiplam is under FDA review. Some speculate a determination could come sometime in May. Regardless of the timeline anticipation is growing. Namely because this is the first oral medication to treat SMA. Because of this I for one will be watching with bated breath for the announcement. I’ve posted about the struggles with spinal injections. My personal feeling is there will be several benefits to a medication you can take by mouth on a daily basis. Of course there are other factors to consider when choosing a treatment. It still blows my mind there will be a choice. It wasn’t too long ago zero treatments were available. Soon there may be three. How will the oral aspect influence your decision whether or not to seek this treatment? What other aspects will you consider? I’m looking forward to discussing it with my neurologist.
March 12, 2020 at 11:45 am #22683Alyssa SilvaKeymaster
I, too, am blown away by the advances taking place in the SMA community. They say good things happen in 3’s, so this can only mean positive changes, right? 🙃
As of right now, I’m going to stick with Spinraza for a few reasons:
1. I haven’t run into too many issues with the lumbar punctures. Yes, it’s nerve-racking and painful, but right now it’s working well for me, so why rock the boat?
2. I have a LOT of GI issues and am just not willing to introduce a new medicine to my body right now. My GI issues are debilitating so I have to be super careful with any medication I take. For example, I cannot take antibiotics because I’ll be sick for months. That’s how sensitive my stomach is, so I’m hesitant to try new drugs.
3. I was the first adult to receive Spinraza, and in some ways, it added unnecessary stress to my life. All eyes were on me, and while I was so fortunate to be in a position where I could be a beacon of light for others, it added some pressure to my world.
That being said, risdiplam isn’t totally off the table for me. I’m going to closely monitor it and be open to the new possibilities. I’m just in no rush.
March 12, 2020 at 9:33 pm #22691Halsey BlocherParticipant
For me, the fact that it’s an oral medication is huge factor! Spinal injections are out of the question for me, but I shouldn’t have any problems with an oral medication. I can just put it through my feeding tube (and therefore also don’t have to worry if it tastes bad). The other big factor in my decision to pursue it is that it doesn’t seem to have any negative side effects. I’ve asked everyone, and no one knows of any.
March 16, 2020 at 2:33 pm #22726Michael MoraleKeymaster
Risdiplam, in my opinion, is going to be a major player within the SMA community. Even though it will work on the SMN2 gene, similar to Spinraza, the opportunity that it provides those who are unable to take Spinraza due to medical conditions, could give these individuals the ability to start on a viable treatments for SMA.
Some of the advantages of risdiplam will be:
- It will be an oral treatment versus an intrathecal injection.
- There will be no hospitalization involved in the dosing of risdiplam.
- Patients who agree to take risdiplam will be getting a dosing every day, versus an injection every 4 months.
- Quite a few patients who are on Spinraza, experience weakness and/or a slight lethargic feeling as they get closer to their next injection. There are hopes that since risdiplam will be a daily dosing, our bodies may not experience this lethargic feeling, because our bodies will be getting a constant supply of this treatment on a day-to-day basis, versus having to wait for months.
As we get closer to the decision from the FDA regarding risdiplam, we may start seeing more information being released, but usually, the FDA restricts the amount of information that can be released until the FDA approves or denies the treatment.
- This reply was modified 3 weeks ago by Michael Morale.
March 19, 2020 at 3:48 pm #22790Chelsea ChandanaisParticipant
If Risdiplam is approved, I plan to switch. I have gotten spinal headaches after every Spinraza injection and the last two required blood patches. Before my last injection, the radiologist said that if I still got a spinal headache despite him trying an atraumatic needle, he would try not removing any CSF next time. Even if I don’t get a spinal headache with my next injection in a few weeks, I can’t continue to deal with the pain of the actual injections every four months (lidocaine has no effect on me and morphine and versed don’t help much) if I have another option that is pain-free.
March 20, 2020 at 9:35 am #22814Alyssa SilvaKeymaster
I’m so sorry the lumbar punctures have been troublesome for you, Chelsea. Hopefully, risdiplam will be the solution for you. It does sound promising.
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