tonight, the cost of saving a life. one child's battle against a rare and deadly disease. >> i noticed she really wasn't moving her legs. her legs did not move at all. >> will the most expensive drug in the world give her a fighting chance? just how much will insurance companies cover? >> it says amount billed $6,193,647.16. >> a family's race against time to give their daughter a future. >> does it feel weird to you to put a pricetag on something that seems priceless? >> this special edition of "nightline," "the price of a miracle," will be right back.

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"nightline," the price of a miracle, continues. here now juju cheng. >> there's no sweeter sound in the world. a melody of pure joy. the sound every parent loves to

hear. but for carrie and rory devine tose precious moments with their daughter reese interrupted. by the sound every parent dreads. the kind that hurts to hear. >> what's the matter with us today, reese? why are you so sad? >> i just can't figure out what's wrong with her today. >> reporter: this laid-back california mom and dad suddenly on a frantic search for answers, trying to unravel a medical mystery sitting right in front of them. and the answer could save countless other babies. >> i was holding her on my lap and just started crying and like saying i'm sorry, i'm sorry. >> reporter: why would you say i'm sorry? >> you just can't help but feel like it's -- like it's our fault.

you know? >> reporter: a rare genetic ase exposing cracks in our medical system. >> hi, donna. it's carrie devine. >> reporter: examining why sometimes there's a jaw-dropping cost to saving a life. >> $6,193,647.16. >> reporter: using the most expensive drug in the world. does it feel weird to you to put a pricetag on something that seems priceless? it might be hard to remember but world reese devine was born into on the eve of 2020 was perfectly normal. just like carrie's pregnancy. what was in your heart when you held her for the first time? >> you're scared. you're excited. you're nervous. but overall you're overjoyed. >> reporter: six pounds, blue

eyes. ten fingers, ten toes. >> you're so big. >> reporter: carrie and rory, like so many first-time parents, falling in love while getting the crash course in life with a newborn. >> oh, my god! >> reporter: what they did not know, what they had not yet come to suspect -- >> hi, sweet stuff. >> reporter: -- was that their beautiful growing baby girl was already losing ground. and to make everything worse, just three months after reese was born covid-19 changed everything. >> there's no comparisons for children because of the pandemic. >> the only other kids we saw were on instagram, and i would get frustrated. i'd be like why is that baby-sitting up? and you know, he or she is two months younger than reese. >> reporter: reese's doctor diagnosed low muscle tone and put her in physical therapy. but instead of getting better, things kept getting worse. >> i noticed she really wasn't moving her legs.

her legs did not move at all. >> consistently hearing from the doctors, oh, just low muscle tone, slow development. you know, she'll get to her milestones. don't worry. >> you pick her up and she slips right through your fingers because she's got no muscle tone. it's like holding a 19-pound newborn. >> oh, baby girl. >> reporter: day after day reese lost ground. and day after day carrie searched for answers. finally getting reese an in-person exam on may 27. that day the pediatrician suspected a new diagnosis. three letters. s.m.a. >> the first thing when you look it up on google is most infants diagnosed with s.m.a. type 1 do not live past 2 years of age. and i was just -- >> 95% mortality rate by the age of 2. >> i just cried. i said i can't -- i can't lose her. like if we lose her i'm -- i don't know what i'll do. >> reporter: s.m.a. stands for spinal muscular atrophy, a

genetic disease passed on only when both parents are carriers p. it affects the central nervous system, specifically the motor neurons in the spine, which affects all the muscles in the body. oer time thaez motor neurons weaken and die off, causing muscles to lose strength. >> people with s.m.a. lose the ability to walk, to sit up even, to swallow and to breathe. >> reporter: carrie and rory ended up at ucla with neurologist dr. perry shay, who confirmed the diagnosis. how come it's so tricky to diagnosis? >> typically these babies when they're born they look essentially normal. >> i think pediatricians are often fooled because these babies are bright-eyed, engaging. they smile at you. they look at you like they know something that you don't. >> reporter: reese not only had s.m.a., she had one of the most severe forms. >> when we first got the diagnosis and we weren't sure if she was going to be alive, i had almost come to terms with the fact, well, this will destroy us. you know, no couple can survive

the loss of their first kid. >> you know, every now and then like we'll go for walks and i'll see like p kids running around or on their scooters and it kind of hits me a little hard because i know that she'll probably never do that. >> what do you think, go back to sleep for mama? >> reporter: luckily there are new treatments, but they're incredibly expensive. >> once you got the diagnosis, what was the race against the clock like? to get the treatment. >> we had started to notice she was declining pretty quickly. >> reporter: the first thing they tried, a drug called spinraza, the first treatment ever approved for s.m.a. >> it sent a lightning rod through the neuromuscular community. having a treatment was a total game changer. >> reporter: each injection, which goes right into the spine, costs about $90,000. and a patient can take them every four months for the rest of their lives. those injections bought reese precious time. while they waited to qualify for a new breakthrough drug. >> unfortuately, it's the most

expensive drug in the world. >> reporter: it's called zolgensma. it targets the genetic root of s.m.a., that missing or mutating gene. it delivers a corrected version, helping the motor neurons function properly. the one-time dose costs $2 million. >> i said to rory, we'll do whatever it takes to get it. i said i would sell my organs -- >> immediately went to organ selling. carrie went -- immediately went to we're selling our organs. and i was like what are you talking about? >> the cost of it didn't really matter. whatever it took we were going to get our daughter this medication. >> reporter: zolgensma is only for kids under the age of 2. the thing is the earlier you get it the better it works. it's so good that if a baby's treated right after birth they could live a relatively normal life. >> the timing of the diagnosis is critical. if s.m.a. is suspected, it is an emergency to figure that out. >> reporter: carrie and rory are both working full-time. both recognize that they have the privilege of health

insurance. still the approval process is complicated and time-consuming. >> do you know about how long it will take? because i think -- >> i spent just all day every day on the phone. i'm like, my baby needs this medication. >> reporter: carrie's desperate and relentless appeals win out. >> crazy. like she has no idea that tomorrow's like the biggest day of her life. >> we got some good news today. reese's treatment has finally been fully approved. >> we still don't know what the final cost is going to be on our end. >> good night, little reese. now close your sweet eyes. >> reporter: it's been 65 days since her official diagnosis. reese is now eight months old. >> now i just have to sleep. which will be the hardest part of this entire thing. >> probably won't be getting much sleep tonight. >> hi, sweetheart. good morning.

are you excited for today? >> reporter: the new day dawns. tinged both with optimism and nerves. >> we're here, reesy. are you ready to go? she looks nervous. >> reporter: the family checks into ucla for the zolgensma injection. >> hey. >> starting to get a little nervous, maybe it's not here. i expected to arrive in a cooler made of diamonds. >> right now it's in a brown bag. >> oh, okay. >> reporter: it's got to be worth its weight in gold. >> it was the largest syringe i'd ever seen. like comically large. oh, boy. it's a big guy. >> it's a big syringe.

>> once they started it and you could see like the syringe depressing it was just like the biggest weight lifted off my shoulder. both of our shoulders. you know, we were just so grateful that she was injection process. reese takes it all in stride. >> i love you too. >> reporter: now it's time to go home. and wait to see if it works. up next -- >> show me how you turn around again. whoa! >> reporter: parents demanding to know why every newborn baby isn't tested for this dangerous disease.

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as the original slice but only a little bit smaller. just like timmy here. my name's lucas. it sure is bobby. "nightline," "the price of a miracle" continues. here again, juju cheng. >> reporter: in addition to blocks and snack time, this is little rhys devine's daily routine. >> rhysy! how are you doing, rhysy? >> reporter: ceri and rory's california home is littered with toys and medical equipment. the effects of having a baby with s.m.a. the rare genetic disease slowly killing the motor neurons in their daughter's spinal cord. >> you're such a brave girl. you didn't even cry when they put the iv in.

>> reporter: they're now waiting to find out if the most expensive drug in the world, worth $2 million, will help save her life. >> it's a couple days after infusion, and rhys is just having a crummy day. and it just sucks. and i just feel bad that i can't help her. >> reporter: were you nervous that it might not work? >> i don't know if we were nervous that it wouldn't work. i never kind of let myself think that. >> reporter: as the couple waited to find out if zolgensma worked for rhys, ceri continued to document the highs and lows on social media. >> look at you go, rhys. >> reporter: she also relied on a facebook group for s.m.a. parents. >> my name is erin, and i'm a mother to emma. she is a 4-year-old with type 1 spinal muscular atrophy. >> my husband and i have two kids. >> our daughter hayden has

spinal muscular atrophy. >> two of my children were born with s.m.a. type 1. >> diagnosed with s.m.a. type 1. >> reporter: many of these parents had no idea what s.m.a. was until it happened to their child. >> my daughter was born before any treatments were available, and she was neve able to sit, stand, walk or talk. >> i wish that we never knew what it was and that this wasn't our story. but it is our story. >> reporter: because time is so crucial, many are now calling on states to make s.m.a. testing mandatory at birth. >> there has been such an enormous advancement in treatment over the past three years. >> we hope that newborn screening becomes the norm in all 50 states because it will give children a much better life than they otherwise would have. >> reporter: in recent months their mission has been gaining traction. nationwide a number of states adding mandatory screening for s.m.a. still, 15% of newborn babies are

not tested. california enacted its mandatory testing six months after rhys was born. >> i just think back to the doctor telling me that you don't have anything to worry about, you're less than 1% chance of anything happening. i mean, that happens. >> it's just so frustrating that now that there's treatment if this was caught when rhys was born her life would look a lot different now. >> reporter: during this time the couple also started learning the real cost of the drug. and rhys's hospital treatment. >> i mean, it clearly says at the top this is not a bill, thank god. but it says amount billed, $6,193,647.16. >> reporter: the drug alone, zolgensma, costs $2 million. why is it so pricey? novartis, the company behind zolgensma, says it only costs about half of what a family would otherwise spend on treatments over a decade. as far as what families actually pay, the company says nearly all its u.s. patients are covered by

insurance. when did you start seeing those tantalizing signs that wow, this thing's working? >> by day three she actually lifted her head up. >> oh, my goodness! >> okay. so why are you crying? >> well, today rhys has started sitting unassisted. and this is just -- it's really a big deal. i'm just really grateful. >> reporter: i've seen video where she turns over from her side onto her back for the first time, and i hear you guys rooting her on and celebrating. >> rhysy! >> i mean, anytime she does anything it's a celebration. you know, i feel like we want to throw a party. >> reporter: they did get to throw rhys a party. her first birthday, pandemic style. ♪ happy birthday dear rhysy ♪

just the three of them. ♪ happy birthday to you ♪ >> she's made some wonderful gains. she's lifting her arms up more than when i first met her. she's lifting her legs up more. she's breathing a bit better.i' >> come and see what the doctor's doing here? >> reporter: by the beginning of 2021 -- >> look at you. >> reporter: -- rhys had gained some new mobility thanks to her first wheelchair, made out of those popular bumbo seats. >> good job. >> do you feel like she'll graduate from wheelchairs someday? >> realistically, she'll probably have a wheelchair as like her primary mode of getting around. it's my hope that she'll be able to walk and take steps and have some independence. >> reporter: that hope may be closer to reality than ceri realized. rhys is learning what it means to be on her own two feet. thanks to special bouncers, shoes, and leg braces.

>> there she goes. >> can you do it again? >> reporter: there's also a specialized machine that helps her stand for longer periods of time. zbl >> good job! >> reporter: but the biggest milestone happened at the end of february, when rhys stood all by herself. for three whole seconds. totally worth it. >> that was good, though, rhysy! >> what are your hopes and dreams for rhys? >> just hope that she stays as happy as she is now. >> my uncle caught me one time when i referred to it as a tragedy and he stopped me and said it's a challenge, not a tragedy. don't forget that. so that's something i have to remind myself all the time. rhys is such an amazing little kid that i think this kind of happened for a reason. >> and just have to keep telling myself that's why we were chosen to be her parents, because you know, we're going to give her the best life possible.

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