Thanks Piton. I don't know how others feel, but this is the but that stood out to me:
"Participants will be randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period."
I don't imagine taking your child to the hospital to recieve a lumbar puncture for what could turn out to be a placebo, is going to be a massive hit among parents/caregivers. Personally, I'd rather a simple, oral dose of something. Now, if only there was a company that had a compound like that in the works...
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