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Re: McMagyar post# 93200

Saturday, 02/25/2017 10:09:44 AM

Saturday, February 25, 2017 10:09:44 AM

Post# of 517507
Rett is a rare disease so finding right patients at place is not an easy task. They need 60 patients and trial duration is 12 weeks (3 months)

This means - the end of the year timeline may prove to be optimistic - for this to happen - the last patient needs to be enrolled before Aug - give one month for topline result calculations/ preparation.

The trial has not started yet - assuming the trial starts by Mid-March and 1st patient is enrolled in April - they will need to enroll 12 patients per month

This number is high for a rare disease trial enrollment and is aggressive.
(They will also compete with other trials for patients)

So if we get topline results by end of year - it is very good sign.

We just need to confirm that protocol is approved and trial is registered and officially started.
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