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This topic has 6 replies, 4 voices, and was last updated 8 months, 3 weeks ago by Alyssa Silva.

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    • #22622
      Alyssa Silva
      Keymaster

      For those of you interested in pursuing risdiplam, have you talked to your neurologist or primary care physician in regard to whether or not they are going to be able to prescribe it to their patients?

      If so, could you please provide some helpful questions or topics to discuss? As of right now, I plan to stay on Spinraza and observe how Risdiplam plays out for adults. But it is still a definite consideration for the future. Would love to see your input.

      Note: risdiplam is the first oral treatment for spinal muscular atrophy awaiting FDA approval. For more information about this treatment, you can get all the details here.

    • #22663
      Julie L
      Participant

      Alyssa – we were just at our MDA clinic visit.  Our neurologist is trying very hard to get Risdiplam now so when it is approved, we can hit the ground running.  The adult MDA clinic hospital would not approve Spinraza so the adults here were basically out of luck.  There is a Pain Clinic facility that started administering to adults, but I have not heard how it’s going.  My 2 adult “kids” with Type II started Spinraza in Dec 2018.  My daughter had to quit after the 4 loading so she is anxious to get on the oral med.  My son is still receiving Spinraza at Children’s, but honestly, we have not seen ANY remarkable improvements but is maintaining.   He just received his 8th dose last month and has actually begun to feel weaker so he too would like to switch to the new drug.   Spinraza just might not work for everyone.

      As for risdiplam – our neurologist said you need to be off of Spinraza for 4 months before you can begin the new drug.  So if our son wants to switch, she said he would need to skip his next dose in May and could not begin the oral med until late summer.   Not sure if that’s the true fact on how it will work, but will find out closer to the date once the drug is approved.

    • #22675
      Halsey Blocher
      Participant

      I am fully intending to start on Risdiplam as soon as possible. My doctors are not anticipating any problems getting it prescribed for me. (Knock on wood.) My hospital is compiling a list of patients who weren’t able to receive Spinraza to get them on Risdiplam quickly. It’s not a long list, but I am on it. So far, I really haven’t heard any negatives, so I’m very hopeful.

    • #22676
      Alyssa Silva
      Keymaster

      Julie, I’m sorry to hear that Spinraza hasn’t been the most effective on your children. I’m sure that must be a little frustrating. Hopefully, that won’t be the case with risdiplam.

      I was told by my neurologist the waiting period between a Spinraza dose and taking risdiplam is 3 months. So it must be somewhere in that 3-4 month time frame. Sending you all positive energy for the process ahead.

    • #22677
      Alyssa Silva
      Keymaster

      You must be so thrilled— and anxious, Halsey!! I’m excited FOR you! Hopefully risdiplam exceeds your expectations. 🙂

    • #22692
      Robert Stump
      Participant

      I have an appointment with my neurologist on Tuesday to talk to him about trying to get on the “Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)”.  This program was announced early in February and the goal seems to get people (who need it sooner rather than later) access to Risdiplam before FDA approval.  It is technically called a study, but the criteria for participation seems pretty broad and it appeared like the company is trying to get more evidence to present to insurance companies if/when the FDA finally does approve it.  It is open for SMA Type 1 & 2 who are not currently able to receive other treatment for whatever reason, and who (in the opinion of the prescribing neurologist) would benefit by receiving the medication quicker.

      My neurologist told me that there is a ton of paperwork for him to submit to allow his facility to participate in this program, but he assured me that he is going to try to get me signed up with it.  I considered Spinraza about a year ago, and almost went through with it, but my breathing issues were concerning to my neurologist and I got the feeling that he wasn’t completely comfortable with me going through it.  At the time I was finally scheduled to start the injections, the hospital where the injections were to take place had a breathing scare with another individual with SMA and they postponed all future injections until they could retrain all of their staff on how to operate various breathing machines someone may arrive to the appointment with.  By the time they wanted to get me back in, I started hearing about Risdiplam and I thought I might as well wait since I was super nervous about the injections anyway.  Hopefully, I made the right choice, but now with the coronavirus potentially being a stumbling block to even go to my appointment safely on Tuesday, I’m not so sure.

      Anyway, for anyone else who is interested in asking their neurologist to see if they can get early access as well, refer to the following website: https://clinicaltrials.gov/ct2/show/NCT04256265

    • #22698
      Alyssa Silva
      Keymaster

      Robert, I was on the Expanded Access Program for Spinraza and am so fortunate for the opportunity. I hope they grant you early access as well. Best of luck to you.

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