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InTheTrenches

04/19/17 9:50 AM

#3642 RE: InTheTrenches #3639

Rhett's syndrome (with Bryostatin):

Pre-clinical work for Rhett's started last May, with funding from Rettsyndrome.org.

My guess is we are within a few months of announcing a Phase 2 trial to commence using Bryostatin. No need to run a Phase 1 trial for safety, as that's already done. Perhaps a quick proof-of-concept trial leading into a double-blind placebo-controlled Phase 2b trial. Or, who knows, maybe NTRP would be bold and jump right into a double-blind placebo-controlled Phase 2b trial.

It affects 1 in 10,000-20,000 females in the US, so it would qualify for orphan drug designation.

If the phase 2b trial is designed correctly, might even get Accelerated Approval?