Ok, it was only a small trial and unblinded, BUT…
Good safety and tolerability – better than trofinetide.
Solid effect – broad and consistent in effect; high statistical significence – better than trofinetide.
Effect clearly seen by both clinicians and caregivers – more so than trofinetide.
Clinically meaningful, as evidenced by clinician and caregiver comments.
Especially impressive result given patients were only on full dose for 7 weeks.
Very pleasing.
Now we can relax!
Ann: P2 trial shows significant improvements in Phelan-McDermid, page-78
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