Share Price, page-3046

Agreed. I work as a doctor in Melbourne and have had several Rett’s Syndrome patients. They are often on multiple aperients to counteract chronic constipation which is associated with the disorder. Taking Trofinetide regularly will hopefully mean the girls need less aperients and are less likely to be admitted with constipation.

In terms of diarrhoea “unblinding” the study, I am not sure how this could be overcome with a repeat phase 3 study. As others have pointed out, most cns drugs have GI side effects, which is a marker that they’re effective. The lack of an advisory committee can only be construed as a positive sign.

I suspect that Trofinetide will be approved in early March. The possible benefits clearly outweigh any harms (mild/moderate diarrhoea, which may actually be advantageous in this patient group).