Thanks for the words about the MD! My father passed away a few years ago from it but my brother (carrier) has no symptoms yet in his 20s. There is no current treatment but more then a dozen companies and billions of dollars have moved into the gene therapy/small molecule/RNA pre-clincal stages in the past 5 years alone with several entering phase 1 in 2021/2022. I'm very hopeful that my brother will have at least one therapy open to him as he starts experiencing symptoms, to the point where he may not be effected at all long term. I'm almost positive he wont have to deal with it much later in life it just depends how much damage is done before a treatment is available. Much like hairloss, the prevention will be easier then restoring atrophied muscles.
And oh no I fully agree, in the other thread about length until new treatments/cures I openly estimated we are about 15-20 years away from full "curative" gene treatments that could prevent all loss, but I do think we are under 10 years away from a new treatment branch where unlimited native hair can be cultured/cloned and either transplanted or administered locally.
First of all I hope your brother and you avoid the MD. Fingers crossed mate.
But I would like to touch upon this discussion as well.
I've been following Parkinson research space for the past 6 months and it seems like an apt comparison for multiple reasons.
PD is not a disease it is more of a syndrome that gathers multiple different causes together because they all lead to similar symptoms.
There are several variants of PD the idiopathic one is the one that happens to most older people (above 50), however there are multiple variants that happen due to multiple possible mutations on gene or genes that are grouped together (PARKIN, PINK1, GBA, SNCA etc.). So basically as you both mentioned those variants are polygenic diseases and would require multiple edits which is definitely a difficult task.
Why did I use this comparison? Because just like Androgenetic Alopecia finding the exact cause of PD (all of the variants) eludes us. This is why I like to compare both.
Thankfully in the case of Androgenetic Alopecia we don't really need to find the culprit. We just need to clone hair follicles and transplant them. Basically I agree with you. We are 15-20 years away from the gene route. However we are definitely close to the cloning part. The timeline will be much more clear when a company like Stemson/ Tsuji/ Hewitt starts their clinical trials.
However when it comes to the new age of medicine and fixing polygenic diseases by fixing genes themselves the cure is still far away.
But that doesn't mean we are not closer to actually treating those diseases. For example a PARKIN PD variant might be due to the oxidative changes causing a certain PARKIN protein to become insoluble. However instead of fixing the genes causing the oxidation of the protein we could supply the soluble version of protein making that variant a treatable disease instead. This is not a cure but just making neurodegenerative diseases treatable would a massive step forward.
To get back to Androgenetic Alopecia one could say that it is already treatable but all of us are here because we know that the current treatments are not good enough. They are just slowing the progression and for a lot of people on this forum not sufficiently or with too grave of side effect profile.
Better treatments might be upon us! SM here might be something like that. I am eagerly awaiting Phase 3 results.