dekhn 1 day ago

Yes, but the cures here aren't general. They're highly specific, and the rare conditions have a long tail- large numbers of different conditions, each with a very small population of affected individuals, and likely, the treatments will be somewhat customized for each type of disease.

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pfisherman 1 day ago

See my comment above. Getting approval for rare diseases and expanding the indication to the common form of the disease is a well established strategy in pharma.

dekhn 1 day ago

yes, but that's totally different from coming up with a generalized treatment for a wide range of "rare" diseases.