SMA News Forums Forums Treatments and Research Zolgensma (onasemnogene abeparvovec) Lottery on free doses of Zolgensma

  • deann-r

    December 20, 2019 at 10:07 am

    Thanks for posting this.  Although this isn’t an ideal solution I do feel like it’s a step in the right direction.  In a perfect world everyone who needed it would have access.  Despite this program expanding access I think there will still be hurdles and of course 100 treatments isn’t nearly enough.  It is better than nothing though.

  • alyssa-silva

    December 20, 2019 at 3:10 pm

    That’s an interesting approach. Not sure if it’ll cause some conflict, but like DeAnn said, it’s better than nothing I guess!

  • adela-mohanu

    December 20, 2019 at 5:40 pm

    Thanks for your inputs.
    My opinion is that the approach is unethical. There are children who don’t have access to any kind of treatment or that have severe side effects frum current treatments. They just might lose the lottery to someone who is already thriving with other available meds. Of course every parent will want to have access to gene therapy, absolutely nothing to blame here. But should someone without access to treatment die because he/she didn’t have so much luck at a draw? That’s a question the company should answer, not the parents.

  • lupa-f

    December 20, 2019 at 9:46 pm

    A doctor has to submit the patient for the program. If the child is thriving under some other treatment, their doctor shouldn’t submit them for this program.

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