One reason PBT2 may not show benefit in motor symptoms is that the patients recruited didn't have motor symptoms.
For raptor and RP103 the motor score had to be greater or equal to 5 to be recruited.
You can't fix a problem people don't have.
Does anyone know if they mentioned whether patients had motor symptoms prior to enrolment as it wasn't an inclusion criteria.
bar for success for pbt2 in hd is very low, page-6
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