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Ann: RP11 Drug Candidate Progressing to Mid-Stage Human Trials, page-10

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    I don’t think that’s quite right. Each drug has to be proven safe. It’s just with ADOA there are multiple other conditions related to the OPA1 gene. They have extra features like deafness or eye muscle weakness. I think these are probably less common than ADOA but I haven’t checked the prevelance.

    It seems that Glaucoma also has an OPA1 mutation. This is a common condition but a quick google (not a proper search) suggests that it may be only the normotensive group that have the mutation (about 50 percent of Glaucoma). Of these it is unclear how many have the OPA1 mutation although I’m sure with a more thorough search we could find out.

    So once proven safe we could use the same drug for all of these conditions so they may all be able to enter phase II together. If a significant proportion of glaucoma are affected this drug will dwarf all the others combined.
 
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