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So, Sairei
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1

α-L-iduronidase fused with humanized anti-human transferrin..:

Harmatz, Paul ; Giugliani, Roberto ; Martins, Ana Maria...
Molecular Therapy.  32 (2024)  3 - p. 609-618 , 2024
Link: https://doi.org/10.1016/..
 
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2

Analysis of caregiver perspectives on patients with mucopol..:

Nakamura, Kimitoshi ; Sakai, Norio ; Hossain, Mohammad Arif...
Orphanet Journal of Rare Diseases.  19 (2024)  1 - p. , 2024
Link: https://doi.org/10.1186/..
 
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3

Efficacy and safety data (52-week) from a phase 1/2 trial a..:

Giugliani, Roberto ; Harmatz, Paul ; Martins, Ana Maria...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107852 , 2024
Link: https://doi.org/10.1016/..
 
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4

A global phase III study of pabinafusp alfa (JR-141) for ne..:

Ikeda, Toshiaki ; Harmatz, Paul ; So, Sairei...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107893 , 2024
Link: https://doi.org/10.1016/..
 
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5

Integrated long-term efficacy and safety data on enzyme rep..:

Eto, Yoshikatsu ; Giugliani, Roberto ; Nakamura, Kimitoshi...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107820 , 2024
Link: https://doi.org/10.1016/..
 
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6

Real-world data of enzyme replacement therapy with pabinafu..:

Eto, Yoshikatsu ; Nakamura, Kimitoshi ; Sakai, Norio...
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107093 , 2023
Link: https://doi.org/10.1016/..
 
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7

International online survey of fucosidosis: Key symptoms an..:

Hamauchi, Kohtaro ; Eisengart, Julie B. ; Paisley-Dew, Carolyn...
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107137 , 2023
Link: https://doi.org/10.1016/..
 
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8

Long-term neurodevelopmental changes in subjects with MPS I..:

Giugliani, Roberto ; Eto, Yoshikatsu ; Nakamura, Kimitoshi...
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107116 , 2023
Link: https://doi.org/10.1016/..
 
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9

Interim results of a phase 1/2 study of JR-171 (lepunafusp ..:

Harmatz, Paul ; Giugliani, Roberto ; Martins, Ana M....
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107140 , 2023
Link: https://doi.org/10.1016/..
 
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10

Changes in quality of life reflecting neurobehavioral impro..:

Martins, Ana Maria ; Mendes, Carmen Silvia Curiati ; Curiati, Marco A....
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107222 , 2023
Link: https://doi.org/10.1016/..
 
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11

Treatment of Neuronopathic Mucopolysaccharidoses with Blood..:

Sonoda, Hiroyuki ; Takahashi, Kenichi ; Minami, Kohtaro...
Pharmaceutics.  14 (2022)  6 - p. 1240 , 2022
Link: https://doi.org/10.3390/..
 
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12

Long term efficacy and safety of pabinafusp-alfa (JR-141) i..:

Giugliani, Roberto ; Martins, Ana M. ; Okuyama, Torayuki...
Molecular Genetics and Metabolism.  135 (2022)  2 - p. S48 , 2022
Link: https://doi.org/10.1016/..
 
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13

A phase I/II clinical study of intravenous administration o..:

Hamazaki, Takashi ; Kawashima, Satoshi ; Ikeda, Toshiaki...
Molecular Genetics and Metabolism.  135 (2022)  2 - p. S52-S53 , 2022
Link: https://doi.org/10.1016/..
 
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14

Phase I/II clinical trial design for a novel therapy for mu..:

Higurashi, Ryo ; Okazaki, Mika ; Kawashima, Satoshi...
Molecular Genetics and Metabolism.  132 (2021)  2 - p. S49 , 2021
Link: https://doi.org/10.1016/..
 
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15

Therapy for mucopolysaccharidosis type II with an intraveno..:

So, Sairei ; Harmatz, Paul ; Kobayashi, Minako...
Molecular Genetics and Metabolism.  132 (2021)  2 - p. S102 , 2021
Link: https://doi.org/10.1016/..
 
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