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Enzyme replacement therapy
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Phase 2
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recombinant human arylsulfatase A
(TAK-611) / Takeda
adrulipase alfa
(FW-EPI) / First Wave Bio
Pertzye
(pancrelipase) / Chiesi
recombinant human heparan N-sulfatase
(SHP610) / Takeda
SB-913
/ Sangamo Therap
GLM101
/ Glycomine
JR-441
/ JCR Pharma
SOBI003
/ SOBI
UX053
/ Ultragenyx, Arcturus Therap
pegtarviliase
(AGLE-177) / Aeglea
CAN103
/ CANbridge Pharma
N-sulphoglucosamine sulphohydrolase
(DNL126) / Denali Therap
