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How different generations living with SMA can teach one another

Meet Regina and Al. Their friendship and respect for one another all started at a fundraiser before a Phillies baseball game in 2019. “I watched Regina give a beautiful speech at the event about her recently diagnosed son, Shane,” recalled Al. “I thought to myself, ‘My friends at Cure SMA need to meet this amazing mom and get her involved.’”


This topic has 4 replies, 4 voices, and was last updated 7 months, 2 weeks ago by Lupa F.

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    • #21786
      Adela Mohanu
      Participant

      So Novartis made it clear: there will be a lottery for the 100 doses it offers out of USA. What’s your opinion about it?

      https://investors.avexis.com/news-releases/news-release-details/avexis-community-statement-global-managed-access-program-for-avxs-101

    • #21788
      DeAnn R
      Keymaster

      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.

    • #21790
      Alyssa Silva
      Keymaster

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

    • #21791
      Adela Mohanu
      Participant

      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.

    • #21794
      Lupa F
      Participant

      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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