Dinakar Singh

And with the FDA as an example, that really helped.

They worked with us and we said, look, guys, we need this to move fast.

And so what can we do?

And by the way, kids are dying.

We can't afford to run a placebo trial.

It doesn't make any sense.

And so we worked with them and got their approval to essentially run a natural history study, meaning while the drugs were being developed, we basically paid for people's care for a handful of years so that we could have a log of what the natural progression of the disease was and use that as our placebo.

And so when it came time for the trials, the trials didn't have to be placebo-avoidant trials, essentially because the FDA was willing to accept the fact that they'll compare the results in the trial to what were the natural history study results of kids normally with SMA.

That saved a lot of lives and a lot of money because otherwise half the kids would have died in those trials, including potentially our daughter.

With all the persistence and with a few little help from our friends as well, the first estimated drug, I think, was then and maybe still now the fastest approval by the FDA ever in history of any drug.

which is pretty cool.

The good news, bad news, of course, my daughter ended up being on the first strike since she was 12.

So it's been stable since then and has ended up being just the rock star of the family.

Went to Yale, won three of the 11 awards for most outstanding student, class speaker, you name it.

And during COVID, amazingly, went on Hinge and met up with this unbelievably amazing 64 fantastic Princeton kid.

They fell in love and they got married last fall.

She's now getting her PhD at Cornell.

So I guess we'll end up with our doctor in the family.

But just watching her thrive has been remarkable.

That said, the clock was the clock.