◼ TECHNOLOGY Bloomberg Businessweek August 26, 201919MARGAUX:
PHOTOGRAPH
BY
MELISSA
GOLDEN
FOR
BLOOMBERG
BUSINESSWEEK.
LYRICK:
PHOTOGRAPH
BY
ANNICK
SJOBAKKEN
FOR
BLOOMBERG
BUSINESSWEEK
THEBOTTOMLINE GenetherapiessuchasZolgensmacarry
unprecedentedpotential—andcosts.Patients,families,andthe
medicalindustryareadjustingin fitsandstarts.were full. Around Thanksgiving, Harlan was scroll-
ing through the posts in a Facebook group when
she saw one from a father who’d been able to
get the drug through Novartis’s compassionate-
accessprogram,theoneforwhichToraPatgiri
didn’tqualify.Childrenintheprogramhadtoshow
symptomsorbediagnosedwithType1 SMA, the
most severe form of the condition, before they were
6 monthsold.Harlanimmediatelycontactedher
neurologisttosecureapproval,gettingthefinalnod
fromthedrugmakeronJan.2. Lucy received the
therapy soon after.
Because she got Zolgensma through the drug
company program, Lucy continues to take Spinraza,
putting her in a new category of children getting
combination therapy. Earlier this summer she did
her first full roll, and she’s starting to lift herself into
the crawling position. “We’re starting to see pretty
remarkable changes,” Harlan says.MEDICAID
MaggieMoore’sexperiencegettinggenetherapy
forherdaughter,Margaux,hasbeenoneofthe
moststraightforward:TheU.S.government’s
health-caresystemcoveredit.
Moore,a stay-at-homemominBirmingham,Ala.,
andherhusband,Alex,a restaurantsous-chef,saw
anabruptdeclineinMargaux’smovementabouta
monthafterherbirthinAugust2017. They figured a
quick call to the pediatrician would reassure them,
but the doctor sent Margaux to the emergency
room, where a neurologist said, before any formal
testing, that she had all the symptoms of SMA.
It took about a month to confirm the diagnosis
with genetic testing. Although Maggie wanted to
enroll her daughter in a study of Zolgensma, there
were no open trials available, so at 4 months old,
Margaux started Spinraza, covered by the federally
subsidized state children’s health insurance pro-
gram. There were signs the treatment was work-
ing: She could sit for brief periods alone and stand
with support, though she still had trouble swallow-
ing and needed help breathing at night.
The day Zolgensma was approved, Moore
was on the phone with her doctor at Children’s
Hospital in Birmingham asking for access to the
treatment. They immediately applied for coverage
from Alabama’s Medicaid program, which covers
Margaux because SMA is considered a disability.
In July, a month before Margaux turned 2, they got
approval for her treatment. There were no hurdles
to jump, only paperwork to complete.
“I had a perception that government insurance
was less proactive,” Moore says. “They have been,
from the beginning, amazing.”WRESTLING WITH THE CHOICE
In Vinton, Iowa, Rani Hopkins struggledwiththe
decisiontogetZolgensma forhersonLyRick,
despiteitsbeinghailedasa miracleanda cure.He
wasborninNovember2017, and she sawproblems
with how he was holding his hand by January.She
took him to the doctor, who said LyRick hadanele-
vated heart rate. He ended up in the hospitaland
later with a neurologist who told Rani andherhus-
band, L.C. Cannady, that their son had “perfectmus-
cle tone” and there was nothing wrong withhim.But
doctorsdon’talwaysfigureit alloutonthefirstvisit.
Twoweekslater,LyRick’sconditionwasundeniable.
“Mysonwentfrombeingwhatneurologistscalleda
perfectly normal child to a limp baby,” Hopkinssays.
LyRick was diagnosed with SMA and givenSpinraza
through Medicaid a few days later.
He’s done so well on Spinraza that Hopkinsis
reluctant to change his regimen, eventhoughit
requires a lifetime of expensive treatments,not
just one shot. Now a few months shy of 2yearsold,
LyRick rolls faster than most people walk,hetalks,
and he sits up unassisted. “I’m fighting withmyself
over this decision,” Hopkins says. “What ifit doesn’t
work? And we can’t get Spinraza? And hedies?”
She has little timefordebate.OnceLyRickis 2,
Medicaid, like other insurers,is unlikelytocover
gene therapy. �CynthiaKoons and MichelleCortez▲ Cannady and Hopkins
with LyRick