Dinakar Singh

Well, actually, technically, he called my wife.

30 seconds, sorry, HSMA, got to go click.

And she called me in tears.

We called him back and it was maybe 60 seconds this time.

But at that time, no one knew much about this disease.

The gene for it was just discovered a few years before.

And until then,

People thought it was just a variant of muscular dystrophy or some other muscle disease.

So in SMA, you're missing a key gene that makes key proteins.

That protein is like a fertilizer for muscles and nerves.

And like fertilizer with plants, without it, they wither and die.

All of us have a backup gene because it's such a critical protein.

Your body has some redundancy.

Some people have one.

Some people have two or three.

That backup gene doesn't work very well.

It makes a protein that falls apart because it's got a defect in it.

But it has some.

And so depending on how many backup genes you have and how little or more than a little protein you have, you might die within a few weeks or you might die within 10, 15 years.

Our daughter was in the middle.