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  • Scott Inouye replied to the topic Risdiplam in the forum Diagnosis​ ​Information​ ​and​ ​General​ ​Questions 7 months, 1 week ago

    My granddaughter is 3 yrs old type 2 and was diagnosed shortly after Spinraza was approved. She has had several treatments now and has seen significant gains. With new drugs being approved and studied, is there anybody looking at these in combination with Spinraza?

    • Scott – Thanks for your question. This is actually a question that’s being asked by many people, especially those of us that are on treatment with Spinraza. I have been on Spinraza now for over 2 years, and like your granddaughter, I’ve seen some improvements, and I think this is a combination of both the Spinraza treatments and taking physical therapy. At this time, the only other FDA approved treatment for SMA is Zolgensma, and the label states that this treatment is for any infant or toddler 2 years of age or younger, irregardless of which type of SMA they have. (Types 1 Through 4).

      Roche/Genentech, along with PTC Therapeutics and the SMA Foundation, will probably be seeking FDA approval for their potential treatment known as risdiplam. This treatment will work on the SMN2 gene, similar to Spinraza, and while there have been rumors that a combination therapy of risdiplam and Spinraza might be in the works, nothing official has been said from either the pharmaceutical company or the FDA. At the present time, our best guess would be that we would hear something regarding risdiplam between the 1st and 2nd quarter of 2020, but again, this is speculation on my part.

      I promise that when we hear something, we will definitely let everyone know on the SMA forum. Please feel free to let me know if you have any further questions, and have a blessed day.

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