00:00:04.260 --> 00:00:07.620
- We got our first snow
of the year in Alabama.

00:00:07.620 --> 00:00:09.930
We don't get to see a lot
of snow, do we?

00:00:09.930 --> 00:00:13.290
So we're gonna go out and play
in it. Let's go. Let's go.

00:00:13.290 --> 00:00:14.850
See how pretty it is!

00:00:16.320 --> 00:00:18.885
What do you think?

00:00:20.465 --> 00:00:22.623
You got mama.

00:00:22.623 --> 00:00:25.045
She's so
excited. And we are too.

00:00:25.045 --> 00:00:28.145
I got ice on my (finger).

00:00:28.145 --> 00:00:29.575
- Wow.

00:00:29.575 --> 00:00:31.555
Lemme take a picture of you.

00:00:31.555 --> 00:00:33.150
Take a picture of you too, baby.

00:00:34.110 --> 00:00:35.676
Oh, you're gonna make a snow angel?

00:00:35.676 --> 00:00:38.273
Yeah. Yeah. Make a snow angel.

00:00:38.273 --> 00:00:40.913
Kick your legs out and your arms.

00:00:43.770 --> 00:00:45.470
- Here we go. Maddie.

00:00:47.661 --> 00:00:48.494
Here we go.

00:00:59.665 --> 00:01:02.850
It's a nice day to be out,
Maddie. 'cause it's sunny.

00:01:02.850 --> 00:01:04.341
We go everywhere.

00:01:04.341 --> 00:01:06.591
Concerts, downtown, everywhere.

00:01:07.680 --> 00:01:09.835
That's what a mom does. You
figure it out and you do it.

00:02:15.005 --> 00:02:17.685
- I'm Celia Grace Hamlett
and I'm five years old.

00:02:22.085 --> 00:02:23.268
I can do it.

00:02:24.676 --> 00:02:28.465
I live in Alabama  with my family

00:02:28.465 --> 00:02:29.957
- Caught a catfish.

00:02:32.305 --> 00:02:34.045
- You can see the little fishes.

00:02:35.370 --> 00:02:37.643
- I love playing with Celia Grace.

00:02:37.643 --> 00:02:38.976
I'd rather hang out with her

00:02:38.976 --> 00:02:40.434
sometimes more than my brothers.

00:02:42.109 --> 00:02:44.659
There's a fish in here
as big as Celia Grace.

00:02:44.659 --> 00:02:45.492
It's a carp.

00:02:47.465 --> 00:02:48.773
- Oh, here we go.

00:02:48.773 --> 00:02:49.915
- Did you see that, Mom?

00:02:49.915 --> 00:02:50.748
- Yeah I did!

00:02:52.915 --> 00:02:54.390
- Mom push me please.
- Okay.

00:02:56.010 --> 00:02:58.260
I'm Kassie Hamlett.

00:02:58.260 --> 00:02:59.435
- Push me higher!

00:02:59.435 --> 00:03:01.576
- It's a good swing, isn't it baby?

00:03:01.576 --> 00:03:03.725
- Colin, come swing with me.

00:03:04.600 --> 00:03:06.460
We live in Winfield, Alabama.

00:03:07.330 --> 00:03:10.630
We're about 80 miles west of Birmingham.

00:03:12.880 --> 00:03:14.558
We're a small town.

00:03:15.520 --> 00:03:18.161
It's very family oriented here.

00:03:18.161 --> 00:03:21.550
♪ Amazing grace. . . ♪

00:03:21.550 --> 00:03:23.500
I'm married to Gary Hamlett.

00:03:24.400 --> 00:03:26.890
We have together five children.

00:03:27.910 --> 00:03:31.670
- This is a big stick.
I can even pick it up.

00:03:31.670 --> 00:03:33.400
- You know, it seems like everybody knows

00:03:33.400 --> 00:03:34.840
who Celia Grace Hamlett is.

00:03:34.840 --> 00:03:36.765
It's kinda like she's a celebrity,

00:03:36.765 --> 00:03:38.698
but not the way you want
her to be a celebrity.

00:03:43.781 --> 00:03:45.864
(chickens clucking)

00:03:50.514 --> 00:03:52.372
(gate opening)

00:03:57.580 --> 00:04:01.900
- My name's Colin
and I'm nine years old

00:04:01.900 --> 00:04:04.150
and I'm Celia Grace's brother.

00:04:04.150 --> 00:04:07.725
- Is that Henrietta?
- Want pet her?

00:04:12.505 --> 00:04:15.400
I'm nervous for her because I'm afraid

00:04:15.400 --> 00:04:17.110
something's gonna go around.

00:04:17.110 --> 00:04:21.550
Celia Grace, let's slide down
the slide. Be careful. Ready?

00:04:21.550 --> 00:04:24.220
She has a rare genetic disease
that infects the brain.

00:04:25.300 --> 00:04:27.725
- Ready?
- I'm scared. I'm scared.

00:04:27.725 --> 00:04:28.558
- It's okay.

00:04:32.345 --> 00:04:36.450
- She is the queen around the house.

00:04:36.450 --> 00:04:38.590
She is the baby and she is the only girl

00:04:40.300 --> 00:04:41.770
and she is our life.

00:04:53.715 --> 00:04:55.681
- Well, she's never had a haircut before.

00:04:55.681 --> 00:04:56.706
Not even a trim.

00:04:57.610 --> 00:04:59.285
Ready to go in and see everybody.

00:05:06.945 --> 00:05:09.670
A mix between maybe not that long,

00:05:09.670 --> 00:05:11.811
but maybe not that short, you know?

00:05:11.811 --> 00:05:12.679
- I like that.

00:05:12.679 --> 00:05:14.297
What all she's been through already,

00:05:14.297 --> 00:05:15.272
she's just scared.

00:05:19.065 --> 00:05:22.180
- I'll make you a princess.
It's like a princess cut.

00:05:22.180 --> 00:05:24.310
- She'll have to take chemo.

00:05:24.310 --> 00:05:28.630
She'll be real sick.
To see her lose her hair.

00:05:30.010 --> 00:05:33.793
That part's gonna be bad.
Just it's gonna be hard.

00:05:33.793 --> 00:05:35.084
- Daddy!

00:05:35.942 --> 00:05:38.075
- Want me to hold you?

00:05:38.075 --> 00:05:40.658
- Okay, come on. I'll hold you.

00:05:42.301 --> 00:05:43.766
It's okay.

00:05:43.766 --> 00:05:44.832
- I'm scared.

00:05:49.447 --> 00:05:53.260
Pull it down. Let it go. Let go. Ooh!

00:05:53.260 --> 00:05:56.500
I talked to my friends about
bringing their little ones

00:05:56.500 --> 00:05:59.300
because I knew if she saw
them, that would help.

00:06:15.770 --> 00:06:18.770
Gary's on the Winfield Police
Department here in town.

00:06:20.390 --> 00:06:21.530
(police radio sounds)

00:06:22.555 --> 00:06:25.760
I'm sitting here at the PD
downloading these pictures

00:06:25.760 --> 00:06:28.880
and body cam from the death this morning.

00:06:31.340 --> 00:06:35.780
I've been a officer about 18
years going on my 19th year.

00:06:35.780 --> 00:06:37.700
This is where Kassie works.

00:06:39.650 --> 00:06:43.730
Our community comes together
in a time of crisis,

00:06:43.730 --> 00:06:46.421
especially when it's dealing
with a child.

00:06:46.421 --> 00:06:47.750
(radio beeps)
Go ahead.

00:06:47.750 --> 00:06:48.725
- Possible 10-89.

00:06:50.289 --> 00:06:54.680
(sirens)

00:06:59.420 --> 00:07:02.030
- She sees me taking care of other people

00:07:02.030 --> 00:07:04.007
and taking care of their problems.

00:07:04.007 --> 00:07:06.769
I look at her knowing that

00:07:06.769 --> 00:07:08.270
(radio chatter)

00:07:08.270 --> 00:07:10.250
I cannot fix her problem.

00:07:11.195 --> 00:07:14.993
- Morning, guys!

00:07:14.993 --> 00:07:19.220
When I was little, I had
gallbladder taking out.

00:07:19.220 --> 00:07:23.885
They said you have
to stay in the hospital.

00:07:23.885 --> 00:07:24.885
I like ice cream.

00:07:28.310 --> 00:07:31.368
- She started having tummy issues.

00:07:31.368 --> 00:07:33.560
- I have everything need.

00:07:33.560 --> 00:07:34.880
And we ended up at the ER.

00:07:37.490 --> 00:07:42.045
- Now you see me, now you don't.

00:07:43.195 --> 00:07:47.810
I had surgery. And they gave me an

00:07:47.810 --> 00:07:50.210
X-ray on my belly button.

00:07:52.265 --> 00:07:54.050
It looks like a picture.

00:07:54.050 --> 00:07:57.260
- The doctor that done
the surgery come out

00:07:57.260 --> 00:07:59.072
and talk to us, but he also said the

00:07:59.072 --> 00:08:00.563
- They gave me an x-ray

00:08:00.563 --> 00:08:01.688
- Okay. Shhh.

00:08:01.688 --> 00:08:03.164
- Go and tell them.

00:08:03.164 --> 00:08:04.370
- And he said

00:08:04.370 --> 00:08:08.090
inside her gallbladder
was a five centimeter mass.

00:08:08.960 --> 00:08:13.340
And they wanted to send it to
pathology for further studies.

00:08:17.900 --> 00:08:21.830
- She came onto my radar
through an email chain.

00:08:21.830 --> 00:08:24.270
The pathologist who was
reviewing the case

00:08:24.270 --> 00:08:26.630
saw something unusual in the gallbladder.

00:08:27.620 --> 00:08:30.943
- He told us it could be
linked to the disease MLD,

00:08:30.943 --> 00:08:33.167
metachromatic leukodystrophy.

00:08:33.167 --> 00:08:35.727
Took me forever
to learn how to say that.

00:08:35.727 --> 00:08:37.440
So of course I did what
you're not supposed to do

00:08:37.440 --> 00:08:38.960
on the way home.

00:08:38.960 --> 00:08:40.193
I Googled.

00:08:47.210 --> 00:08:49.910
It read that they don't
live to be five years old.

00:08:52.040 --> 00:08:55.032
- It affects the central
nervous system.

00:08:55.032 --> 00:08:58.110
Difficulties remembering things.

00:08:58.110 --> 00:09:00.510
They will lose the ability to walk.

00:09:00.510 --> 00:09:02.393
They will lose speech.

00:09:02.393 --> 00:09:03.459
There is no cure.

00:09:03.459 --> 00:09:04.734
And these children die.

00:09:08.700 --> 00:09:11.580
- And I just lost it. And Gary did too.

00:09:12.690 --> 00:09:14.193
We cried all the way home.

00:09:15.060 --> 00:09:16.860
I said, what are we gonna do, Gary?

00:09:18.450 --> 00:09:21.030
- I don't show a whole
lot of motion around people.

00:09:23.473 --> 00:09:25.048
I had a breakdown.

00:09:25.890 --> 00:09:27.540
- We're gonna lose her. That's just all

00:09:27.540 --> 00:09:28.620
that we had in our mind.

00:09:28.620 --> 00:09:29.620
We're gonna lose her.

00:09:32.670 --> 00:09:34.230
- She didn't have any evidence

00:09:34.230 --> 00:09:39.060
of changes in her reflexes in
terms of her neurologic exam.

00:09:39.060 --> 00:09:40.830
We never have this situation

00:09:40.830 --> 00:09:43.830
where we have a basically
asymptomatic child.

00:09:46.680 --> 00:09:49.260
None of us really knew
how much time she had

00:09:49.260 --> 00:09:51.000
before she started to develop symptoms.

00:09:51.000 --> 00:09:54.600
We just knew that at this
moment in time, she had none.

00:09:55.740 --> 00:09:59.250
There is this problem in her genes

00:09:59.250 --> 00:10:01.620
and you're just waiting
for the other shoe to drop.

00:10:18.750 --> 00:10:22.590
- And now Cory's gonna
do the the morning meds.

00:10:22.590 --> 00:10:23.610
- Her morning meds.

00:10:35.160 --> 00:10:38.970
- I adopted Maddie from China
when she was 13 months old.

00:10:38.970 --> 00:10:41.550
They brought her in the room.
I recognized her immediately.

00:10:41.550 --> 00:10:43.433
And this was my daughter.

00:10:44.374 --> 00:10:46.590
She had the cutest little voice.

00:10:46.590 --> 00:10:51.231
- I am going to get you my pretty.

00:10:51.231 --> 00:10:53.797
- My pretty! (laughs)

00:11:03.030 --> 00:11:07.530
- Maddie was diagnosed with
metachromatic leukodystrophy.

00:11:07.530 --> 00:11:10.260
She was stumbling when she walked.

00:11:10.260 --> 00:11:12.690
And then later she had
issues with swallowing.

00:11:12.690 --> 00:11:14.850
- You taking a nap, Miss Maddie?

00:11:17.550 --> 00:11:20.220
- As it progressed, she
had trouble with breathing.

00:11:21.886 --> 00:11:24.286
- Cheese! (laughs)

00:11:28.980 --> 00:11:31.038
- Scratch my head, would ya?

00:11:31.038 --> 00:11:32.910
- Not me.

00:11:32.910 --> 00:11:34.170
- Do you feel like you need more suction?

00:11:34.170 --> 00:11:35.863
Close your eyes if you need more suction.

00:11:35.863 --> 00:11:37.857
Close your eyes if you
need suction in your mouth.

00:11:42.475 --> 00:11:45.360
- We moved from Ohio to Minnesota

00:11:45.360 --> 00:11:48.450
to a doctor here that
knew the rare disease.

00:11:49.980 --> 00:11:53.031
And they did a bone marrow transplant.

00:11:53.031 --> 00:11:55.407
♪ Silent night. ♪

00:11:55.407 --> 00:11:57.160
When we came back after a hundred days,

00:11:57.160 --> 00:11:58.270
they had to do it again.

00:11:59.470 --> 00:12:00.586
They had to figure out

00:12:00.586 --> 00:12:02.052
how much can this little body take?

00:12:05.530 --> 00:12:06.363
Is that okay?

00:12:08.838 --> 00:12:10.946
She has a little feeding pump over here

00:12:10.946 --> 00:12:12.820
that runs 24-7

00:12:12.820 --> 00:12:16.330
and then twice a day we use green beans,

00:12:16.330 --> 00:12:17.590
stage two baby food.

00:12:17.590 --> 00:12:19.135
And we mix it in with the formula

00:12:19.135 --> 00:12:20.385
to help her get some more

00:12:20.385 --> 00:12:22.270
of the dietary needs that she has.

00:12:22.270 --> 00:12:24.190
- Her job is to hold onto her feeding tube

00:12:24.190 --> 00:12:25.900
so it doesn't get caught on anything.

00:12:25.900 --> 00:12:28.210
So you got that, Mad?

00:12:31.300 --> 00:12:34.480
- I think the bone marrow
transplant definitely helped.

00:12:34.480 --> 00:12:37.478
It took its toll too
because of the damage

00:12:37.478 --> 00:12:39.100
that chemo and radiation do.

00:12:40.390 --> 00:12:42.131
And at that time, that's the
only option

00:12:42.131 --> 00:12:44.410
that or do nothing.

00:12:44.410 --> 00:12:47.740
- All right. Mad. You
ready? Ready to rumble.

00:13:03.550 --> 00:13:05.982
- Now I'm going to color my dad.

00:13:07.545 --> 00:13:09.405
He has a fat belly.

00:13:12.875 --> 00:13:15.646
And a long big head.

00:13:15.646 --> 00:13:17.860
And his eyes.

00:13:17.860 --> 00:13:21.218
And it let color my mommy.

00:13:21.218 --> 00:13:23.526
She's right here.

00:13:25.870 --> 00:13:28.990
- It's heartbreaking because

00:13:28.990 --> 00:13:32.250
me and Kassie has talked about it,

00:13:32.250 --> 00:13:34.480
she's like that because of us.

00:13:35.440 --> 00:13:38.350
- Our genetics doctor, she
drew out this whole thing.

00:13:38.350 --> 00:13:40.180
And of course you
learned in science class,

00:13:40.180 --> 00:13:41.860
you know, dominant recessive.

00:13:41.860 --> 00:13:44.260
If you have too much of
something, it could be bad.

00:13:46.210 --> 00:13:48.901
- There is this particular enzyme

00:13:48.901 --> 00:13:51.205
called Arylsulfatase Alpha

00:13:51.205 --> 00:13:52.930
or ARSA.

00:13:52.930 --> 00:13:56.350
And the person that has
metachromatic leukodystrophy

00:13:56.350 --> 00:14:00.730
has a problematic change on
both copies of that gene.

00:14:01.720 --> 00:14:05.080
Then there is buildup
of a toxic substance.

00:14:05.080 --> 00:14:07.383
That is what results in cell death.

00:14:14.680 --> 00:14:16.690
- I'll color this right here.

00:14:17.890 --> 00:14:19.870
I'll color pink clouds.

00:14:24.820 --> 00:14:28.360
- This great hope was a gene therapy trial

00:14:28.360 --> 00:14:30.460
that was happening only in Italy.

00:14:40.510 --> 00:14:41.510
- At that time,

00:14:41.510 --> 00:14:44.290
I had done some research on gene therapy.

00:14:46.390 --> 00:14:48.070
- It was a lot to take in.

00:14:48.070 --> 00:14:50.200
It was kind of like way over my head.

00:14:51.770 --> 00:14:53.420
- With gene therapy,

00:14:53.420 --> 00:14:57.440
what happens is Celia's
own stem cells are taken

00:14:57.440 --> 00:15:02.360
and they use the properties
that viruses have to infect us

00:15:02.360 --> 00:15:06.967
and they insert a functioning ASRA gene

00:15:06.967 --> 00:15:08.870
into that virus.

00:15:08.870 --> 00:15:11.420
And then when they put the genes back,

00:15:11.420 --> 00:15:13.190
as her body starts rebuilding,

00:15:13.190 --> 00:15:16.610
it starts copying the good gene

00:15:16.610 --> 00:15:18.626
- Here I've told them that

00:15:18.626 --> 00:15:21.560
at some point in time in the future,

00:15:21.560 --> 00:15:24.500
their daughter will have
neurologic symptoms.

00:15:24.500 --> 00:15:26.750
She will start to go backwards.

00:15:26.750 --> 00:15:27.680
- Get it!

00:15:27.680 --> 00:15:28.910
- and then I come back to them

00:15:28.910 --> 00:15:31.880
and I say, okay, maybe
you have to go to Italy.

00:15:32.780 --> 00:15:35.120
- We can't do that. We can't afford it.

00:15:35.120 --> 00:15:37.370
We can't go over there
for four months and live.

00:15:37.370 --> 00:15:38.395
We can't do it.

00:15:38.395 --> 00:15:41.030
Now pull it back. Pull it back.

00:15:41.030 --> 00:15:43.250
Oh, you got it.

00:15:43.250 --> 00:15:46.160
- We're talking about early 2020.

00:15:46.160 --> 00:15:48.154
In the beginning of the pandemic

00:15:48.154 --> 00:15:50.504
when Italy's ground zero.

00:15:52.850 --> 00:15:54.654
- I was like, wow. In Italy?

00:15:54.654 --> 00:15:56.900
I'd never flown before

00:15:56.900 --> 00:15:58.940
and I was a nervous wreck.

00:15:59.960 --> 00:16:04.610
- Couple weeks later,
Dr. Ananth called Kassie.

00:16:04.610 --> 00:16:06.470
- I remember the phone call.

00:16:06.470 --> 00:16:08.450
She said, what if I told you

00:16:09.470 --> 00:16:12.193
that your daughter was
gonna make history books?

00:16:12.193 --> 00:16:15.380
Perhaps I'm that dramatic,
I don't know. (laughs)

00:16:15.380 --> 00:16:16.730
- She said, your daughter is going

00:16:16.730 --> 00:16:20.270
to be the first child in the
United States to be treated

00:16:20.270 --> 00:16:23.420
with gene therapy for MLD.

00:16:23.420 --> 00:16:24.440
- And that's a big deal

00:16:24.440 --> 00:16:26.660
because it could
potentially clear the path

00:16:26.660 --> 00:16:29.120
for other children in the United States

00:16:29.120 --> 00:16:30.260
to get this treatment.

00:16:34.915 --> 00:16:35.748
(calling to dog) Gage!

00:16:37.655 --> 00:16:38.655
- Ready?

00:16:41.835 --> 00:16:45.457
Come on, Gage! Come on.

00:16:50.060 --> 00:16:52.100
I hope everything goes
well. And she gets better.

00:16:59.660 --> 00:17:01.610
- And we'll be praying for y'all

00:17:01.610 --> 00:17:04.430
and we'll be praying for
Celia Grace's healing

00:17:04.430 --> 00:17:05.690
and y'all's protection

00:17:05.690 --> 00:17:08.270
and just think about the
all the other children

00:17:08.270 --> 00:17:10.220
that eventually are going to be able

00:17:10.220 --> 00:17:12.155
to be helped because of this.

00:17:12.155 --> 00:17:12.988
- Amen.

00:17:12.988 --> 00:17:15.440
- And I know it's a trying time,

00:17:15.440 --> 00:17:17.660
but we just have to keep our faith.

00:17:17.660 --> 00:17:19.010
Father, we just thank you Lord.

00:17:19.010 --> 00:17:21.530
We just pray that you
just give them courage.

00:17:21.530 --> 00:17:24.110
That you would just give them strength.

00:17:24.110 --> 00:17:26.000
And Father, we know there's gonna be days.

00:17:26.000 --> 00:17:27.800
It's gonna be really bad.

00:17:27.800 --> 00:17:30.080
And Father, we just
pray you just lift them

00:17:30.080 --> 00:17:31.285
up and see 'em through it.

00:17:31.285 --> 00:17:35.965
♪ Praise God. Praise God. Praise God. ♪

00:17:39.105 --> 00:17:39.938
- And everybody said?

00:17:39.938 --> 00:17:40.771
- Amen!

00:17:43.388 --> 00:17:45.260
- Celia Grace with her little baby

00:17:45.260 --> 00:17:46.968
watching her iPad.

00:17:46.968 --> 00:17:48.915
What's your cat's name?

00:17:48.915 --> 00:17:51.705
- Wucky.
- Lucky!

00:17:53.310 --> 00:17:57.240
This is something Celia Grace
done for Father's Day.

00:17:57.240 --> 00:18:00.030
This is something that means a lot to me.

00:18:00.030 --> 00:18:02.250
It says, walk with me dad.

00:18:03.463 --> 00:18:04.755
- Say good morning.

00:18:04.755 --> 00:18:08.970
- Good morning. Are we
going to the airplane.

00:18:08.970 --> 00:18:11.610
- Yeah, we're going to the
airport. You ready to go?

00:18:11.610 --> 00:18:12.443
- Yes.

00:18:12.443 --> 00:18:13.890
- We starting our journey today.

00:18:13.890 --> 00:18:15.723
- I'll go get my suitcase.

00:18:15.723 --> 00:18:19.620
- We're headed out to
Minneapolis, Minnesota

00:18:19.620 --> 00:18:24.150
for the beginning of our
journey with gene replacement.

00:18:26.460 --> 00:18:30.000
- Walk alongside of me daddy,
and hold my little hand.

00:18:35.340 --> 00:18:37.350
I have so many things to learn

00:18:37.350 --> 00:18:39.033
that I don't understand.

00:18:40.799 --> 00:18:41.691
Are you nervous?

00:18:41.691 --> 00:18:42.766
- Momma is.

00:18:44.160 --> 00:18:46.493
- Teach me things to keep me safe

00:18:46.493 --> 00:18:48.476
from dangers every day.

00:18:51.420 --> 00:18:54.182
Show me how to do my best

00:18:54.182 --> 00:18:57.220
at home, at school, at play.

00:18:58.230 --> 00:19:00.822
- All checked and carry on baggage

00:19:00.822 --> 00:19:03.505
is subject to search.

00:19:03.505 --> 00:19:06.448
- Every child needs a gentle hand

00:19:06.448 --> 00:19:08.873
to guide them as they grow.

00:19:10.140 --> 00:19:15.122
So walk alongside me, daddy,
we have a long way to go.

00:19:48.480 --> 00:19:52.530
- Hi guys. Nice to meet you.
Welcome to Minnesota.

00:19:52.530 --> 00:19:54.600
I'm sure it's a little bit of a weather

00:19:54.600 --> 00:19:55.980
transition for you guys.

00:19:55.980 --> 00:19:57.646
Yes sir, it is.

00:19:59.513 --> 00:20:01.171
She's asymptomatic

00:20:01.171 --> 00:20:02.435
which is pretty unusual.

00:20:02.435 --> 00:20:05.100
Most of the kids that are
picked up with MLD

00:20:05.100 --> 00:20:07.440
are because they have neurologic problems.

00:20:07.440 --> 00:20:09.544
- I want you to copy what I do, okay?

00:20:09.544 --> 00:20:10.610
Ready friend?

00:20:10.610 --> 00:20:13.710
Look at that. Celia. Awesome job.

00:20:13.710 --> 00:20:16.335
And I've got an animal here.
It is a . .

00:20:16.335 --> 00:20:18.210
- Monkey!
- Monkey.

00:20:18.210 --> 00:20:21.180
- She has not progressed
from a neurologic standpoint.

00:20:21.180 --> 00:20:23.070
Gives us almost a unique opportunity

00:20:23.070 --> 00:20:25.290
and time to be able to intervene

00:20:25.290 --> 00:20:27.330
before she develops those types of issues.

00:20:31.405 --> 00:20:36.120
- We're gonna have an MRI
tomorrow. A lumbar puncture.

00:20:36.120 --> 00:20:38.130
I'm kind of nervous about that.

00:20:38.130 --> 00:20:39.635
- I'm gonna look in your
ear here. Just a second.

00:20:39.635 --> 00:20:41.075
- He's gonna check your ear out.

00:20:44.190 --> 00:20:45.395
- This to Celia Grace's room.

00:20:47.710 --> 00:20:49.390
There's her little Peppa Pig.

00:20:51.970 --> 00:20:54.700
She is back in MRI right now.

00:20:54.700 --> 00:20:56.230
Held her while they put her to sleep

00:20:56.230 --> 00:20:58.360
and then they took her out of my arms

00:21:02.290 --> 00:21:06.490
And we had to leave.
Had to leave my baby there.

00:21:14.740 --> 00:21:16.510
- I've talked to too many families

00:21:16.510 --> 00:21:18.610
that we've had to turn away.

00:21:18.610 --> 00:21:21.100
I'm sorry I, I can't offer your son

00:21:21.100 --> 00:21:24.250
or daughter any effective treatment.

00:21:24.250 --> 00:21:27.460
Go home and spend as much
time as you can with them.

00:21:27.460 --> 00:21:29.560
'cause they're going to get sicker

00:21:29.560 --> 00:21:32.170
and progress and they're gonna die.

00:21:32.170 --> 00:21:34.600
That is just not acceptable.

00:21:34.600 --> 00:21:36.430
And that's what really motivates me.

00:21:36.430 --> 00:21:40.570
We jus t can't live with not
being able to do something

00:21:40.570 --> 00:21:42.877
for these, for these families.

00:21:45.245 --> 00:21:46.861
- Hey, I found something!

00:21:46.861 --> 00:21:48.082
- What'd you find?

00:21:48.082 --> 00:21:49.365
- Sit down.

00:21:49.365 --> 00:21:51.370
- I just found water.

00:21:51.370 --> 00:21:53.478
- That's the Mississippi River.

00:21:53.478 --> 00:21:54.311
Put your hat up.

00:21:55.420 --> 00:21:57.619
- Say, see you later, Minneapolis.

00:21:57.619 --> 00:21:59.219
Say, we'll see you soon.

00:22:02.075 --> 00:22:03.495
- Celia Grace. Tell me who come.

00:22:04.715 --> 00:22:05.875
- Santa Claus.

00:22:05.875 --> 00:22:07.438
- What'd he bring you?

00:22:07.438 --> 00:22:09.796
- Awotta presents!

00:22:16.315 --> 00:22:19.156
- Did you get some stuff
for Christmas, Celia Grace?

00:22:21.035 --> 00:22:23.510
- Celia Grace doing her schoolwork.

00:22:23.510 --> 00:22:24.476
- Look!

00:22:26.425 --> 00:22:28.545
- Merry Christmas, Dad.

00:22:28.545 --> 00:22:31.000
- Merry Christmas Celia. I love you, baby.

00:22:34.360 --> 00:22:37.810
- I made a promise years ago
when I first found out about

00:22:37.810 --> 00:22:40.030
her disease that we were
gonna live life large.

00:22:40.030 --> 00:22:43.180
We were gonna do everything we
could to not feel the impact

00:22:43.180 --> 00:22:44.650
of the disease.

00:22:44.650 --> 00:22:47.050
And so we did a lot of crazy things

00:22:47.050 --> 00:22:48.316
so that she wouldn't feel

00:22:48.316 --> 00:22:50.124
that she was dying every single day.

00:22:51.670 --> 00:22:53.950
Welcome to Maddie's Wall of Men.

00:22:53.950 --> 00:22:56.470
We started with

00:22:56.470 --> 00:22:59.890
appreciating hot men when we
would turn that wheelchair

00:22:59.890 --> 00:23:02.140
however we had to so she
had the perfect view.

00:23:02.140 --> 00:23:04.630
We have firefighters from Florida.

00:23:04.630 --> 00:23:07.240
We have people that we
wish we had in our life

00:23:07.240 --> 00:23:08.860
that we don't, but they're hot.

00:23:08.860 --> 00:23:11.830
And we appreciate that fact.

00:23:11.830 --> 00:23:14.470
This doctor said, why is it,

00:23:14.470 --> 00:23:18.610
I saw a picture when you
did the telehealth visit.

00:23:18.610 --> 00:23:21.626
What are all those men and
why is this doctor up there?

00:23:21.626 --> 00:23:23.585
And I'm not?

00:23:23.585 --> 00:23:26.118
We have Maddie's
favorite bus driver here.

00:23:26.118 --> 00:23:29.890
We have the Schwan's man.
We have her orthotics guy.

00:23:29.890 --> 00:23:31.720
We have another couple doctors here

00:23:31.720 --> 00:23:33.760
that have been a gift to us.

00:23:33.760 --> 00:23:35.500
We have Dr. Paul Orchard, who is one

00:23:35.500 --> 00:23:36.940
of the superstars on our wall.

00:23:37.840 --> 00:23:42.130
When we told him that we needed
a picture for the Man Wall,

00:23:42.130 --> 00:23:43.420
he's like, I don't know.

00:23:43.420 --> 00:23:46.400
And I go, just send us
anything. We'll put it up.

00:23:46.400 --> 00:23:48.124
I'm sorry Paul (laughs).

00:23:52.775 --> 00:23:55.445
- ♪ Happy birthday to you. ♪

00:23:56.895 --> 00:24:01.310
♪ Happy birthday to Celia Grace. ♪

00:24:01.310 --> 00:24:05.450
♪ Happy birthday to you. ♪

00:24:05.450 --> 00:24:06.620
- It was special before,

00:24:06.620 --> 00:24:09.530
but now, thank I God every day

00:24:09.530 --> 00:24:11.530
that I get another day with her.

00:24:11.530 --> 00:24:13.296
What's she doing?

00:24:13.296 --> 00:24:14.780
Look at him, he's scratching his back.

00:24:16.435 --> 00:24:18.218
- Whoa, he's so big.

00:24:18.218 --> 00:24:20.060
- Yeah, they're so big, ain't they?

00:24:20.060 --> 00:24:20.893
- Yeah.

00:24:22.970 --> 00:24:25.105
(Celia Grace screams)

00:24:32.230 --> 00:24:33.547
(Celia Grace screams)

00:24:33.547 --> 00:24:35.072
Daddy! I want my mommy!

00:24:35.072 --> 00:24:37.398
- I'm sorry sweetie. I'm sorry.

00:24:39.305 --> 00:24:40.880
- How do you technically describe it

00:24:40.880 --> 00:24:42.740
to a 4-year-old without terrifying 'em?

00:24:44.720 --> 00:24:47.107
Just look at us. You're fine.
He's just listening.

00:24:48.915 --> 00:24:50.360
- It's okay. You're doing a good job.

00:24:52.250 --> 00:24:53.141
- Excellent.

00:24:53.141 --> 00:24:55.124
Essentially this is how the

00:24:55.124 --> 00:24:57.418
gene therapy process works.

00:24:57.418 --> 00:25:00.740
First, we need to collect blood
stem cells from Celia Grace

00:25:00.740 --> 00:25:03.080
using a process called apheresis.

00:25:03.080 --> 00:25:06.320
- That's it. You've got
this down. Very nice.

00:25:06.320 --> 00:25:08.390
Those cells are then shipped to Milan.

00:25:08.390 --> 00:25:10.430
There the cells undergo engineering

00:25:10.430 --> 00:25:12.440
to produce the missing enzyme.

00:25:12.440 --> 00:25:13.940
See, it's not sharp or anything.

00:25:13.940 --> 00:25:15.260
I'm just gonna look in your ear.

00:25:15.260 --> 00:25:16.709
- That tickles!

00:25:16.709 --> 00:25:18.009
- That tickles?

00:25:18.009 --> 00:25:20.360
- Looks pretty good.

00:25:20.360 --> 00:25:24.560
Next those cells are frozen
and shipped back to us.

00:25:24.560 --> 00:25:26.000
After chemotherapy,

00:25:26.000 --> 00:25:29.000
those cells are infused intravenously.

00:25:29.000 --> 00:25:30.140
These cells will grow

00:25:30.140 --> 00:25:32.960
and provide the enzyme that she needs

00:25:32.960 --> 00:25:35.120
to help stop progression of the disease.

00:25:42.666 --> 00:25:44.316
- It's okay.

00:25:50.812 --> 00:25:52.603
- Can I sit in your lap?

00:25:55.955 --> 00:25:57.204
- Why? (crying)

00:25:57.204 --> 00:25:59.829
- It don't hurt, baby.
No, it don't hurt.

00:26:08.435 --> 00:26:10.876
(crying)

00:26:12.875 --> 00:26:13.708
- Stop.

00:26:15.402 --> 00:26:17.876
- Mama, mama (crying).

00:26:18.835 --> 00:26:20.443
Stop. Stop.

00:26:26.775 --> 00:26:30.830
- She's got a lot of strength.
She's got a lot of will.

00:26:30.830 --> 00:26:31.830
- She's very strong willed. (laughs)

00:26:31.830 --> 00:26:35.745
- Very strong. Very strong-willed.

00:26:35.745 --> 00:26:37.160
- In a lot of stuff.

00:26:37.160 --> 00:26:40.640
- Just seeing her ability to stay strong

00:26:40.640 --> 00:26:43.980
through this was inspiration.

00:26:58.650 --> 00:27:02.463
- It's really hard being in
this space, this house

00:27:02.463 --> 00:27:04.740
with all of her equipment and stuff here.

00:27:06.900 --> 00:27:09.450
I believe I was chosen to be her mom.

00:27:10.830 --> 00:27:12.000
I love being her mom.

00:27:13.770 --> 00:27:14.603
If I could make her laugh,

00:27:14.603 --> 00:27:15.936
it was the greatest gift in the world

00:27:15.936 --> 00:27:17.880
to see her laugh and smile.

00:27:20.485 --> 00:27:22.170
I was trying to teach her to text.

00:27:23.580 --> 00:27:25.650
So I'm holding her little tiny finger.

00:27:25.650 --> 00:27:28.422
And I said, you can text
your boyfriend.

00:27:28.422 --> 00:27:31.050
You can text I love you. Or you're hot.

00:27:31.890 --> 00:27:33.510
She is laughing so hard

00:27:33.510 --> 00:27:37.140
and as I'm holding her, I could feel her,

00:27:37.140 --> 00:27:41.550
you know, just so excited
and having so much fun.

00:27:44.220 --> 00:27:45.840
We didn't hold back on anything

00:27:45.840 --> 00:27:48.960
because it was so important
to get her to laugh every day.

00:28:18.630 --> 00:28:22.770
- This always looks dramatic.

00:28:22.770 --> 00:28:24.240
With smoke coming out.

00:28:30.420 --> 00:28:34.740
- Good morning, gorgeous.
How are you? How'd you sleep?

00:28:36.360 --> 00:28:38.160
Huh? How'd you sleep?

00:28:41.370 --> 00:28:44.880
Today is the big day. She
get her new genes back.

00:28:51.120 --> 00:28:53.125
- How close are we to having cells read?.

00:28:53.125 --> 00:28:55.055
- 15 minutes.
- Oh, 15 minutes.

00:28:55.055 --> 00:28:57.355
It's been a long time coming,
getting to where we are.

00:28:57.355 --> 00:28:59.075
And you know, it takes a village

00:28:59.075 --> 00:29:00.955
to do these types of things.

00:29:00.955 --> 00:29:03.155
So the people involved
in obtaining the cells,

00:29:03.155 --> 00:29:05.455
all the nurses and other physicians

00:29:05.455 --> 00:29:07.235
that were involved in this.

00:29:07.235 --> 00:29:10.140
So it's taken a lot of time and
effort to get to this point.

00:29:10.140 --> 00:29:11.850
But we're excited to be
able to move forward.

00:29:17.310 --> 00:29:19.440
- Oh, this is where I need my glasses.

00:29:22.200 --> 00:29:25.410
So this is OTL dash 200,

00:29:26.790 --> 00:29:30.655
batch 2 1 0 6 9

00:29:32.005 --> 00:29:33.938
dash or slash B?

00:29:33.938 --> 00:29:36.713
OTL 200.

00:29:36.713 --> 00:29:39.795
CU UMN zero one.

00:29:39.795 --> 00:29:40.935
- Yes.
- Okay.

00:29:45.655 --> 00:29:46.655
- There it is.

00:29:52.690 --> 00:29:54.820
- These are blood stem cells
that have been engineered

00:29:54.820 --> 00:29:56.320
to make enzyme.

00:29:58.390 --> 00:30:00.940
- All right. Cells. Onward.

00:30:04.600 --> 00:30:08.100
The expression is "Cells wait for no one."

00:30:18.190 --> 00:30:19.540
- We've been hoping

00:30:19.540 --> 00:30:23.470
and praying for this day
for a year and a half.

00:30:24.940 --> 00:30:27.607
It's just unreal that it's here.

00:30:30.945 --> 00:30:33.778
All right, Celia Grace, we're
done! Yay!

00:30:38.155 --> 00:30:39.738
- Look at her hair.

00:30:41.995 --> 00:30:43.832
- What color is her hair?

00:30:44.875 --> 00:30:45.708
- Blue.

00:30:45.708 --> 00:30:48.733
With my Barbies with me,

00:30:50.470 --> 00:30:53.170
That made me really brave.

00:30:53.170 --> 00:30:54.310
- Here's all your clothes.

00:30:54.310 --> 00:30:57.084
There's the baby blankets.

00:30:57.084 --> 00:30:59.184
Where'd the baby go?

00:30:59.184 --> 00:31:00.851
- Look, Daddy.

00:31:02.230 --> 00:31:05.830
- The bond that I have with her is

00:31:06.940 --> 00:31:10.000
so amazing and I guess

00:31:10.000 --> 00:31:12.070
that's the reason it was so devastating.

00:31:12.910 --> 00:31:17.020
Something so precious
that could be taken away.

00:31:19.480 --> 00:31:24.115
- Dad always be beside me when I'm scared.

00:31:24.115 --> 00:31:25.720
- How's it look? See Grace?

00:31:25.720 --> 00:31:27.610
I'm cutting my hair for you.

00:31:28.900 --> 00:31:31.452
That way Daddy looks just like you.

00:31:32.320 --> 00:31:33.815
Here goes. You ready?

00:31:39.790 --> 00:31:42.400
Me having to leave to come home

00:31:42.400 --> 00:31:45.850
and leave them there so far away.

00:31:45.850 --> 00:31:48.940
That was one of the hardest
things I've ever had to do.

00:31:48.940 --> 00:31:50.768
All right, mama

00:31:55.870 --> 00:31:58.695
- You and daddy are
Twinkies!. Look at y'all.

00:32:07.925 --> 00:32:09.375
♪ Happy happy discharge day. ♪
♪ We wish the best for you. ♪

00:32:09.375 --> 00:32:11.850
♪ Wash your hands. Wear your mask. ♪

00:32:11.850 --> 00:32:15.358
♪ And take a med or two.♪ - Wooo! ♪

00:32:41.635 --> 00:32:43.311
- Come get me!

00:32:43.311 --> 00:32:46.010
Well, we're at Ronald McDonald House

00:32:46.010 --> 00:32:48.650
and I'm just thankful that we're here now

00:32:48.650 --> 00:32:51.350
and that she's doing so well.

00:32:51.350 --> 00:32:53.150
- Can you hold my hand?

00:32:53.150 --> 00:32:54.975
- Come on, I'll hold your hand right here.

00:32:54.975 --> 00:32:57.050
- Weeee!

00:32:57.050 --> 00:33:00.845
- They give me the medicine.
It goes in my tummy.

00:33:02.525 --> 00:33:05.208
- Straight down the middle.

00:33:05.208 --> 00:33:07.657
The things I do for Celia Grace.

00:33:10.688 --> 00:33:12.784
- I like it Colin!

00:33:12.784 --> 00:33:13.635
It like it!

00:33:13.635 --> 00:33:15.543
This what mine looks --

00:33:15.543 --> 00:33:18.017
It looks like mine!

00:33:21.475 --> 00:33:24.980
- We made it a little
better, didn't, we honey?

00:33:26.120 --> 00:33:29.636
Oh. Are you gonna get more
candy than me?

00:33:29.636 --> 00:33:31.597
You look great, Celie.

00:33:31.597 --> 00:33:34.700
Ready? Okay, come on. Let's go.

00:33:35.660 --> 00:33:36.920
We're going together. Let's go.

00:33:39.050 --> 00:33:41.465
- I love your costume.
- Thanks.

00:33:41.465 --> 00:33:43.585
- Look at this

00:33:43.585 --> 00:33:45.005
- You wanna shake my hook?

00:33:46.555 --> 00:33:47.915
- Well, here you are, sweetheart.

00:33:47.915 --> 00:33:49.365
- Say thank you.
- Thank you.

00:34:04.190 --> 00:34:06.410
- This is called TPN.

00:34:06.410 --> 00:34:10.520
It's actually nutrients
that she is getting

00:34:10.520 --> 00:34:12.980
because she's not eating well.

00:34:12.980 --> 00:34:16.430
I was really afraid at
first about having to do it

00:34:16.430 --> 00:34:18.590
because I'm not a nurse.

00:34:18.590 --> 00:34:21.590
I'm just a mom.
You know, I work at a bank,

00:34:21.590 --> 00:34:23.240
but I've learned it.

00:34:26.000 --> 00:34:28.340
So I'll take that off.

00:34:30.200 --> 00:34:33.920
And now, I always say,
Celia Grace calls it milk.

00:34:33.920 --> 00:34:36.178
'cause that's what it looks like.

00:34:36.178 --> 00:34:37.394
She likes to help.

00:34:37.394 --> 00:34:40.730
I let her feel like she's mixing.

00:34:42.020 --> 00:34:43.250
I don't want her to be afraid.

00:34:43.250 --> 00:34:45.230
I want her to know
everything that's going on.

00:34:46.790 --> 00:34:51.620
Her line's like yours.
- I wanna see it.

00:34:51.620 --> 00:34:52.970
- What'd you do with her house shoes?

00:34:54.405 --> 00:34:57.045
- Her house shoes? I don't know.

00:34:57.045 --> 00:35:01.025
- She has this thing.
It just looks like mine.

00:35:03.530 --> 00:35:04.530
- Sit down.

00:35:04.530 --> 00:35:05.363
We're just gonna show her
how we hook up. Okay?

00:35:05.363 --> 00:35:06.500
- No!

00:35:06.500 --> 00:35:07.333
- Sit down for mama.

00:35:07.333 --> 00:35:08.814
- No.

00:35:08.814 --> 00:35:09.647
- Yeah, listen.

00:35:10.555 --> 00:35:11.625
No, I don't to.

00:35:13.580 --> 00:35:14.580
- No ma'am.

00:35:14.580 --> 00:35:16.446
I wanna go home.

00:35:16.446 --> 00:35:18.112
- I know you do.

00:35:19.585 --> 00:35:20.785
I know. It's okay.
You gotta eat.

00:35:22.093 --> 00:35:24.444
But I wanna go home now.

00:35:24.444 --> 00:35:25.908
- I know.

00:35:25.908 --> 00:35:27.170
I know.

00:35:27.170 --> 00:35:29.686
- I wanna go see my brothers.

00:35:29.686 --> 00:35:32.930
- I know. They miss you too.

00:35:32.930 --> 00:35:34.760
What do you think about
Colin shaving his head?

00:35:34.760 --> 00:35:35.593
What'd you think?

00:35:35.593 --> 00:35:36.426
- Hmm, hm.

00:35:42.795 --> 00:35:47.795
- This one. Thank you.
You gonna hold it for me?

00:35:48.450 --> 00:35:49.552
Thank you.

00:35:49.552 --> 00:35:50.385
Catch it!

00:35:52.015 --> 00:35:53.965
- It's so slippery.

00:35:55.945 --> 00:35:57.486
- Hey. Hello.

00:35:57.486 --> 00:35:58.752
- Celia Grace.

00:35:58.752 --> 00:35:59.752
- She's pretty busy over there.

00:35:59.752 --> 00:36:03.760
- Dr. Orchard's gonna
listen to you, okay?

00:36:03.760 --> 00:36:05.190
All right, this side. Is that cold?

00:36:09.030 --> 00:36:11.550
Deep breath for me. That a girl.

00:36:13.080 --> 00:36:14.190
Excellent.

00:36:15.300 --> 00:36:18.600
So a couple weeks ago we
sent that enzyme level.

00:36:18.600 --> 00:36:21.030
Got like 20 to 80 is the normal range.

00:36:21.870 --> 00:36:24.090
And she's like 120.

00:36:24.090 --> 00:36:27.995
So she's actually, her enzyme
levels are above normal now.

00:36:27.995 --> 00:36:28.920
- Good!

00:36:28.920 --> 00:36:30.630
- Oh yeah. That's
what we're trying to do.

00:36:30.630 --> 00:36:31.921
- So, okay.

00:36:31.921 --> 00:36:33.329
- Yeah, that's, that's
kind  of the key thing.

00:36:33.329 --> 00:36:34.704
- That's an improvement.

00:36:34.704 --> 00:36:37.170
- Yes it is. Oh my goodness.
That's so good. Yeah.

00:36:37.170 --> 00:36:38.580
- No, that's exciting.

00:36:38.580 --> 00:36:40.490
- It is. It's very exciting.

00:36:40.490 --> 00:36:42.190
- Is that my brain?

00:36:42.190 --> 00:36:43.380
That's your brain. Yep.

00:36:43.380 --> 00:36:45.390
- They told us they
thought everything took,

00:36:45.390 --> 00:36:48.240
but you know, until those
numbers really show, you know,

00:36:48.240 --> 00:36:51.480
you got this little thought
in the back of your mind,

00:36:51.480 --> 00:36:52.313
did it really take?

00:36:52.313 --> 00:36:54.813
Go give Dr. Orchard a high five.

00:37:08.022 --> 00:37:10.705
(wind chimes and footsteps)

00:37:19.575 --> 00:37:21.360
- You are my rock, Maddie.

00:37:22.890 --> 00:37:25.410
At the celebration of life,
I encouraged people

00:37:25.410 --> 00:37:29.550
not to bring flowers, but to
bring rocks that they painted

00:37:29.550 --> 00:37:31.920
or that they found or
that had a special memory.

00:37:32.910 --> 00:37:35.910
This is so beautiful.
"Owl" always love you.

00:37:45.455 --> 00:37:48.555
And they're from doctors
and friends and relatives.

00:37:49.830 --> 00:37:53.280
One of her teachers -- you'll
always be my favorite student.

00:37:53.280 --> 00:37:57.630
She taught me. That's so cool.

00:38:15.060 --> 00:38:16.230
Love abundantly

00:38:16.230 --> 00:38:19.260
and figure out how can
you make this life better.

00:38:25.170 --> 00:38:28.110
I'm really having a hard
time because I miss her

00:38:28.110 --> 00:38:32.820
and I miss all the beauty and
the fun that we had together

00:38:32.820 --> 00:38:35.230
and all the cool things
that we did together.

00:38:35.230 --> 00:38:38.230
I never felt like it was a burden on me.

00:38:38.230 --> 00:38:41.647
It's what she needed. And she was my kid.

00:38:59.660 --> 00:39:01.820
- I can't stop popping, wheelies!

00:39:05.140 --> 00:39:08.650
I'm really excited.
It's been almost a year.

00:39:08.650 --> 00:39:10.366
I think she looks great.

00:39:10.366 --> 00:39:12.482
I'm gonna do a couple more things.

00:39:12.482 --> 00:39:15.700
Can you touch your nose and then his nose?

00:39:15.700 --> 00:39:19.180
And then your nose. All right my dear.

00:39:19.180 --> 00:39:24.160
Watch this with just your
eyes. Okay. Very good.

00:39:25.915 --> 00:39:29.805
- Quick, Colin! They just laid some eggs.

00:39:33.215 --> 00:39:37.525
- I'm trying to catch him.

00:39:43.260 --> 00:39:44.927
I catched it!

00:39:44.927 --> 00:39:46.630
- Celia Grace's family was

00:39:46.630 --> 00:39:47.950
able to do this.

00:39:47.950 --> 00:39:49.960
Even go to Minnesota.

00:39:49.960 --> 00:39:54.430
I have many patients whose
parents are the most loving

00:39:54.430 --> 00:39:58.360
people who would do
anything for their children.

00:39:58.360 --> 00:40:00.970
That logistically that could not happen.

00:40:04.840 --> 00:40:07.840
- If we better understand
some of these diseases

00:40:07.840 --> 00:40:09.850
and how best to intervene,

00:40:09.850 --> 00:40:13.690
that's got far reaching
consequences in terms of being able

00:40:13.690 --> 00:40:15.880
to move medicine forward.

00:40:21.670 --> 00:40:25.900
- It's why I went into
this field, the hope that I

00:40:26.890 --> 00:40:28.300
get to do this once.

00:40:29.530 --> 00:40:32.890
Yeah. Sorry.

00:40:36.280 --> 00:40:38.950
- And this is that you unicorn Barbie.

00:40:40.565 --> 00:40:45.125
I feel grateful and happy
and excited for every day.

00:40:47.385 --> 00:40:51.095
♪ See love, time heals. ♪

00:40:53.595 --> 00:40:57.620
Take this. So put it right here.

00:40:57.620 --> 00:40:58.453
- I won!

00:40:59.517 --> 00:41:01.885
(laughing)

00:41:01.885 --> 00:41:04.125
(joyful scream)

00:41:04.125 --> 00:41:06.700
- I'm so excited for kindergarten.

00:41:07.915 --> 00:41:09.700
- This is my backpack.

00:41:09.700 --> 00:41:12.280
We definitely made
history books as they say.

00:41:13.210 --> 00:41:15.220
I just hope for her after this.

00:41:15.220 --> 00:41:18.553
I just want her to do
whatever makes her happy.

00:41:26.080 --> 00:41:27.490
- Sometimes we feel guilty

00:41:27.490 --> 00:41:29.890
because we got the chance

00:41:29.890 --> 00:41:32.830
that these other kids may not get.

00:41:35.615 --> 00:41:38.073
- I'm collecting leaves.

00:41:40.373 --> 00:41:42.905
- We found a cute worm!

00:41:42.905 --> 00:41:47.540
My goal is to get this
treatment approved in the US.

00:41:48.650 --> 00:41:52.370
If it saves one child,

00:41:52.370 --> 00:41:54.080
then I feel like we have
accomplished something.

00:41:56.985 --> 00:42:00.830
- The fact that there's now something

00:42:02.090 --> 00:42:04.853
that can save them is a beautiful thing.

00:42:04.853 --> 00:42:07.162
Am I jealous? You bet.

00:42:07.162 --> 00:42:10.100
But is it a great thing? You bet.

00:42:10.100 --> 00:42:11.337
- Heavenly Father we
come to you at this time.

00:42:11.337 --> 00:42:13.811
Father, we ask you to bless
all this food, Father. Amen.

00:42:13.811 --> 00:42:14.644
- Amen.

00:42:14.644 --> 00:42:16.652
Y'all want green beans?

00:42:16.652 --> 00:42:19.152
I'm thankful for Celia Grace being able

00:42:19.152 --> 00:42:23.735
to get the treatment and
thankful for her doing so well.

00:42:27.092 --> 00:42:28.900
- That's me.

00:42:30.968 --> 00:42:32.868
Woo hoo and I'm done!

00:42:40.306 --> 00:42:45.306
♪ See Love by Emily Haavik ♪