Moonsoon Briefing Thurs 20 April, page-65

Good question @Taureanbull Here is my laymans take. Each of those 100+ indications is the result of a different genetic mutation all of which ending up producing a similar range of symptoms. Those symptoms eg hand wringing, poor communication, gastrointestinal problems seem to be improved across the board by our molecules. So it might be difficult for a regulator to sign off on approval for use in all or many indications just because it is efficacious in 4 or 6.

If that was the case a Doctor with a patient with say a Rett's adjacent condition would be busting to prescribe 2591 off label. But how do you get reimbursed for a $300k drug that's not approved specifically for your patient's syndrome?

Maybe a BP with a huge trial budget could design a blockbuster trial for a spectrum of syndromes and symptoms and get to a HUGE market that way. Others here will know bettter than me so feel free to jump in.