SMA News Forums Forums SMA News Today Multimedia Flash Briefings and Podcasts The SMA News Today Podcast 44: Roundtable Discussion October 2019

  • deann-r

    October 15, 2019 at 11:58 am

    It will be interesting to see if the higher dose will be safe and effective. I have to say I felt more effect from Spinraza during the loading doses and through the first maintenance dose. It stands to reason this would be the case because there would’ve been more of the drug in my system at one time. I’m guessing higher dose would be more costly, but hopefully it wouldn’t be something like double the dose double the price.

    Risdiplam is certainly intriguing. I like that since it’s an oral medication it works on a cellular level. As with many I also like that it’s not a spinal injection. With it being a daily medication it may also be a more steady result unlike the ebbs and flows many of us on Spinraza see. My concern of course is cost and pharmacy issues. One of the only reasons I’m getting Spinraza is that the University of Minnesota does buy & bill. I can’t see a regular pharmacy doing that.

    Thankfully I’ve never been sprayed, but I do have skunk stories. For some reason they do like to take refuge under ramps. My sister had to tell me the windows automatically shut if there’s a skunk around just so I could sleep with the window open. Don’t get me started on spiders!

    We could probably start a separate forum on wheelchair stories.

    • kevin-schaefer

      October 15, 2019 at 4:07 pm

      I’m interested too in seeing what kind of effect the higher dosing will have on people in this study. Likewise, I felt a big boost of energy during my Spinraza loading doses. With the maintenance doses, I do feel a slight decrease in stamina when another dose approaches.

      Risdiplam is definitely promising. I just got back from a Cure SMA leadership conference in Boston, and Genentech gave a really good presentation there. Granted, they can’t say much right now about the estimated cost until they get FDA-approval, but it’ll be interesting to see where things go in the next few months.

      And lol, I want to hear these skunk and spider stories!

Log in to reply.