SquirrelOnFire 1 day ago

30 million people in the US are affected by "rare" genetic conditions.

2
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.

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.

pfisherman 1 day ago

Also rare genetic diseases give insight into the underlying mechanisms and pathology of common sporadic diseases, which can be leveraged to develop new and better therapies.

Getting a new drug or therapy approved for a rare form of a disease and then expanding the indication to the common disease patient population is a well established strategy.