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One of the hardest parts is slowly losing your independence and mobility. It is really hard to continually adjust my expectations for my life. It is hard to not feel defeated when I have to keep letting go of activities I used to be able to do. And I’m especially scared of the future because my muscles will only grow weaker without treatment.

– Voice of the Patient Report, submitted to the U.S. Food and Drug Administration (FDA), February 21, 2023