3 ms·
30 million people in the US are affected by "rare" genetic conditions.
by SquirrelOnFire 1y ago
30 million people in the US are affected by "rare" genetic conditions.
- dekhn 1y agoYes, 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 1y agoSee 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 1y agoyes, but that's totally different from coming up with a generalized treatment for a wide range of "rare" diseases.
- pfisherman 1y agoAlso 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.