2019-08-26 Bloomberg Businessweek

◼ TECHNOLOGY Bloomberg Businessweek August 26, 2019

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milestoneswouldcomewithtime.Theydidn’t.

Aftera misdiagnosis,monthsoftherapy,and

scoresoftestsandprocedures,thefamilywas

toldElianahadSMAinJune.Whatshedidn’thave

wastime.Zolgensmaisa potentialcure,butit’s

approvedonlyforchildrenunderage2,beforeirre-

versibleharmis done.Eliana’ssecondbirthdaywas

fiveweeksaway.Afterinsurancerefusedtocover

thetreatment,ShaniandAriel,a rabbi,turnedto

theChesedFund,a crowdfundingorganization

fortheOrthodoxJewishcommunity.

“Asa mother,I havetodoeverythingpossibleto

helpmydaughter,”Shanisaidina videothataccom-

paniedthefamily’sfundraisingplea.“Everything,

evenif it makesmeuncomfortable.”

Sotheyreachedouttostrangers,andit worked.

Morethan23,000peoplefromtheU.S.,U.K.,and

IsraeldonatedforEliana’streatment,mainlysmall

amountsrangingfrom$5to$180.Oneanonymous

donorpledged$285,000tobringthecampaignover

the$2.2milliontarget.It onlytookfivedays.

“Idon’tknowwhatthefuturewillbringfor

her,”Shanisaysinthevideo.“Idon’tknowwhat

machines,whattubes,whatanything.AllI knowis

thatI wantmydaughtertohaveanopportunityto

havea normallife,justlikeeveryotherkid.”

ElianareceivedZolgensmaonJuly19,theday

afterhersecondbirthday.

IMMIGRATING

PortfoliomanagerRajdeepPatgirihasbeenona

crusadeforhisdaughter,Tora.Shewasbornin

theU.K.inSeptember2018.ByJanuaryheandhis

wife,TaisiyaUsova,a stay-at-homemother,noticed

thatshedidn’thavemuchmovement.Hesearched

“babydoesn’tmoveherleg”ontheinternetand

thefirstfewanswerspointedtoSMA.HetookTora

totheemergencyroom,butthedoctorsaidit was

likelya developmentaldelayandsentthemhome.

Patgirikeptpursuingit,andtwomonthslatera

neurologistgavehimthedreadedSMAconfirma-

tion.Therewerenoapprovedtreatmentsforthe

conditionintheU.K.,soheaskedhisemployerif he

couldmovehisfamilytotheU.S.togetToraintoa

Zolgensmastudy.Fiveweekslater,herelocatedto

hiscompany’sNewYorkoffice,thoughhe’sbeen

workingremotelywhilethefamilyhasfocusedon

gettingToratreatmentinColumbus,Ohio.

Battling insurance wasn’t his initial plan.

PatgiritriedtogetZolgensmathroughNovartis’s

compassionate-access program, which makes

experimental drugs available for free while they’re

awaiting regulatory approval. But that was closed to

them because he wasn’t a U.S. citizen or green-card

holder. He applied for Zolgensma in June through

his UnitedHealthcare insurance plan and was

denied within days, because the insurer covered

the drug only for children younger than 6 months.

There was a wrinkle. On June 25, the day

UnitedHealthcaresentPatgirithedenial,it alsopub-

lishednewguidelinesexpandinguseofthedrugto

childrenupto 2 years old. “I actually saw the new

guidelines first, and two hours later I saw the denial,

which didn’t make sense,” he says. The insurer told

him Tora’s case reviewer didn’t have the authority

to approve her treatment. So he shared their story

withtheWashingtonPostandothermediawhilecon-

tinuingtheappealsprocess.A weekafterthestory’s

publicationinnewspapers, Tora was approved. The

family plans to move to New York in January.

COMPASSIONATE ACCESS

Sara Harlan has survivor’s guilt. The social worker

is in the SMA Facebook groups, follows the news,

and knows how much trouble some parents have

had getting Zolgensma. For her daughter, Lucy,

things weren’t quite so complicated, and she says

that weighs on her. “I have a really, really hard time

knowing how easy it was to get this stuff,” she says.

Lucy didn’t move much when she was born in

Louisville in April 2018. She was diagnosed with SMA

when she was 10 weeks old. She started on Spinraza

within days and almost immediately started kicking

her legs. “That was like hitting the lottery,” Harlan

recalls. “We just screamed.” But the doctor had told

her about Zolgensma, and she and her husband,

Danny, an accountant for health insurer Humana,

set their minds on gene therapy. The clinical trials

◀ Moore with Margaux