Dinakar Singh

And it worked and it didn't work.

She did end up needing spinal fusion.

and having rods put on her back, et cetera.

But fortunately, those were late enough that she had already grown, and so it didn't create the deformity you'd normally see in someone else.

She had problems with her hips that blew out and things like that.

When she was turning 10, 11, every year, there became a major surgery.

And even aside from those major surgeries, she was in the operating room, or she was in the hospital probably three times a year with severe pneumonia at risk of dying each time.

And with all these things, it is a bit Humpty Dumpty-like.

Once it breaks, you can't just put it back together again.

On one hand, if it had been a year earlier, that would have been all the better.

But the good news is that compared to what we expected at the outset, to see her here and a rock star and truly happy with amazing friends and husband is just- So touching.

I describe to people, if you have a boat and you have a hole in it, trying to build water out of the boat is not really going to be a useful strategy.

You're going to sink just maybe a tiny bit slower.

Once you fix that goal, and now there's water on the boat, now building water on the boat could actually be a really good strategy.

Mission stage two now is that we're working on a ton of drugs that we think can regenerate muscle.

Nerve is harder to do, but muscle is a bit easier and also help existing nerves get a little bit stronger on stuff.

We have one already in the clinic.

It's not the best one, but we expect to have three or four things coming forward that could be really impactful.

And what's actually pretty cool about this is that

The SMA drugs are SMA-specific.