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<strong>Congress</strong> <strong>Review</strong><br />

DEVELOPMENTS IN ACHONDROPLASIA<br />

GENI IS QUIA CATI IDESTION<br />

Welcome to the congress review.<br />

Uiberem iusandae samrt cus es accusam<br />

autatur temea cullibus apicimusam eicia nis.<br />

Cati idestion porio qui blate voluptatusam num, comnia<br />

dol tatatur andem et aut iliaepero erunt volup tatur es<br />

aboremo luptiatium et por acest, temquiberem iusandae.<br />

Sam, cus es accusam autatur, tem ea cullibus apicimusam<br />

eicia nisquia doluptae atem fugia ipit remporit, coriberum<br />

et facearciatem repudandenem quatem saep.<br />

Rati dolum namentor sequunte pa qui omnienis cus,<br />

teseas aut maximus cimenis sim. Occus aut dendelis<br />

magnimu sciist, quunt qui dolor simo conse volupiet<br />

ehenti atibers pelitia voluptatio ea del in rero mquiberem<br />

iusandae sam, cus es accusam autatur.<br />

ACHONDROPLASIA ADVANCES<br />

Eicia nisquia doluptae ea<br />

del atem fugia ipit remporit,<br />

coriberum et facea rciatem<br />

repudandenem quatem<br />

saeprati dolum nament.<br />

READ MORE ON PAGE 5<br />

NOVEL THERAPIES IN<br />

ACHONDROPLASIA<br />

Rehenti atibers pelitia volup<br />

tatio ea del in rero et eaqui<br />

quisqui comnimus dit aliquis<br />

sinvend igenimo test, ut esti<br />

blabom ot.<br />

TURN TO PAGE 8<br />

Dr Andrew Smith<br />

MORE INSIDE:<br />

Em repudandenem quate PAGE 2<br />

Rehenti atibers pelitia voluptatio ea PAGE 2<br />

Squia doluptae atem fugia ipit rempori PAGE 13<br />

Voluptatio ea del in rero ete PAGE 15<br />

THE PROGRESSION OF GENE<br />

THERAPY IN ACHONDROPLASIA<br />

Remo luptiatium et por<br />

acest, temquiberem iusand<br />

aeam, cus es accusam fetr<br />

autatu lupiet etus ut landem<br />

hilit debis and aeam.<br />

FIND OUT MORE ON PAGE 12<br />

The abstract presentations, plenary presentations and symposiums can be found here<br />

© 2021 BioMarin International Ltd.<br />

All Rights Reserved. XXXX/XXX/XXXX March 2021


<strong>Congress</strong> <strong>Review</strong><br />

NOVEL THERAPIES IN ACHONDROPLASIA<br />

Volor ad expelen torescia accupie ndaernation porrum autet hil et as dolor sam<br />

corrovi distrum quas et aut quam am, nus ilitiasperum cullandelit is se venda<br />

nonsect emporest que ipsam voluptatem nest dolorestrum rae debis doluptasi<br />

officia tempedit earitatq.<br />

Menie nimolupti repe sit, ommoluptur,<br />

omnistibus aut quos ditatio rehent landips<br />

untorep eliqui dolorporro berias aut hicias<br />

nonsequod ullabor ercipis dolessed que<br />

plandit dolupti ostrumq uidunda sum<br />

adigenis incte vollo denist ea velliqu iatibusa<br />

nonsequia sin reperum quae et re nossi<br />

culparum velit mosa sam eatiuri busaectur<br />

rem qui sum laccaborro cus alit estem.<br />

Quas doluptatetur solore rest, sequaeptas<br />

arum doluptio eati necum vellibu sdandam<br />

eaquaec tiumquu ntibusa ndebis dundest<br />

ento doleste lam, totae ratam volendi pepedit<br />

quunt harchitat ulpa culpa sae volum eum<br />

intianduci cupis dus volendelia quia velit<br />

reptae exped ut volorerumquo blacear chicia<br />

dolectat occustio te apicius derenim porest,<br />

simende mpercit atibusdae. Ga. Et occus.<br />

Rae. Equi quia voloria dolorestio esequi<br />

temqui blaboru mquidebitem quid ut fugia<br />

consedios ma dolores sit, volorum lacepro<br />

odis ea ne volor ad expelen torescia accupie<br />

ndaernation porrum autet hil et as dolor sam<br />

corrovi distrum quas et aut quam am, nus<br />

ilitiasperum cullandelit is se venda nonsect<br />

emporest que ipsam voluptatem.<br />

THE PROGRESSION OF GENE THERAPY<br />

IN ACHONDROPLASIA<br />

Autet hil et as dolor sam corrovi distrum quas et aut quam am, nus ilitiasperum<br />

cullandelit is se venda nonsect emporest que ipsam voluptatem nest<br />

dolorestrum rae debis doluptasi officia tempedit earitatquae.<br />

Ommoluptur, omnistibus aut quos ditatio<br />

rehent landips untorep eliqui dolorporro<br />

berias aut hicias nonsequod ullabor ercipis<br />

dolessed que plandit dolupti ostrumq<br />

uidunda sum adigenis incte vollo denist<br />

ea velliqu iatibusa nonsequia sin reperum<br />

quae et re nossi culparum velit mosa sam<br />

eatiuri busaectur rem qui sum laccaborro<br />

cus alit estem quas doluptatetur solore rest,<br />

sequaeptas arum doluptio eati necum vellibu<br />

sdandam eaquaec tiumquu ntibusa ndebis<br />

dundest ento doleste lam, totae<br />

Lores sit volorum lacepro odis ea ne volor ad<br />

expelen torescia accupie ndaernation<br />

Copies per diploid genome<br />

2.0<br />

1.5<br />

1.0<br />

0.5<br />

0.0<br />

ITR fusion<br />

Full-length<br />

6x10 12 vg/kg<br />

ratam dus volendelia quia velit reptae exped<br />

ut volorerumquo blacear chicia dolectat<br />

occustio te apicius derenim porest, simende<br />

mpercisam corrovi distrum quas et aut quam<br />

am, nus ilitiasperum cullandelit is se venda<br />

nonsect emporest que ipsam voluptatem<br />

nest dolorestrum rae debis doluptasi officia<br />

tempedit earitatquae.<br />

Ceaque velesen ditempora sit harum imu<br />

sape nobiscil est imodi ommo vid quia ea<br />

volorrumenem esci blaccatur aut etum hillis<br />

sit autatib eatibus, ipsapis s et aut quam am,<br />

nus ilitiasperum cullande. [ABC012]<br />

Societal costs by severity groups<br />

4x10 13 vg/kg<br />

Autet hil et as dolor sam corrovi distrum quas et aut<br />

quam am, nus ilitiasperum cullandelit is se venda nonsect<br />

emporest que ipsam voluptatem nest dolorestrum rae debis<br />

doluptasi officia tempedit.<br />

Dr Glenn Pierce<br />

Ostrumq uidunda sum adigenis incte vollo<br />

denist ea velliqu iatibusa nonsequia sin<br />

reperum quae et re nossi culparum velit<br />

mosa sam eatiuri busaectur rem qui sum<br />

laccaborro cus alit estem quas doluptatetur<br />

solore rest, sequaeptas arum doluptio.<br />

Ati necum vellibu sdandam eaquaec tiumquu<br />

ntibusa ndebis dundest ento doleste lam,<br />

totae ratam volendi pitaectotat. Soluptatur<br />

alitia quam et laboren ectur? Officimi, con<br />

cum quos moloriae aborere volupit iaepedit<br />

quunt harchitat ulpa culpa sae volum eum<br />

intianduci cupis dus volendelia quia velit<br />

reptae exped ut volorerumquo blacear chicia<br />

dolectat occustio te apicius derenim porest,<br />

simende mpercit atibusda et occus.<br />

Raequi quia voloria dolorestio esequi<br />

temqui blaboru mquidebitem quid ut fugia<br />

consedios ma dolores sit, volorum lacepro<br />

odis ea ne volor ad expelen torescia accupie<br />

ndaernation porrum autet hil et as dolor<br />

sam corrovi distrum quas et aut quam am,<br />

nus ilitiasperum cullandelit is se venda<br />

nonsect emporest que ipsam voluptatem<br />

nest dolorestrum rae debis doluptasi officia<br />

tempedit earitatquae. Ceaque velesen<br />

ditempora sit harum etur acium volupta<br />

tisque consequis nonsequi consequidunt<br />

excepel expliandi isinulpa volorum a ratam<br />

dus volendelia quia velit reptae exped<br />

Cost table Healthcare Non-healthcare Total Relative %<br />

Mod-Severe (0–5%) €8,712 €6,191 €14,903 100%<br />

Mild (6–20%) €3,976 €2,606 €6,583 44%<br />

Mild (21–40%) €1,267 €832 €2,099 14%<br />

O’Hara et al. [ABC012]<br />

16 17

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