According to this
there are around 10 000 rare diseases and only a fraction of them - around 500 can be cured/treated.
Even if the numbers are not correct, generally, the negative prospects of treating rare diseases are well known. In general, there is not much hope for those suffering from an orphan disease.
Even if we find out one day, what went wrong in our bodies on a cellular level, really, we will just become other people suffering from officially recognized rare disease with no treatment, won´t we…? Purely based on these statistics.
I am not trying to spread negative spirit here, but that is the conclusion my rational mind has come to.
Or is there any reason to believe, that our case would be any different from other orphan diseases without treatment? In terms of how difficult it would be to treat PFS.
And another question. Generally, why is that, there are so many diseases without any treatment? Sure, money, but besides that is that because for the vast majority of these rare diseases the real cause has not yet been discovered, or the cause is known, but no treatment has been invented?