E-LIB Suche - Ergebnisse für: Tanizawa, Kazunori

1

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/..

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/..

3

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/..

4

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/..

5

A phase I/II clinical trial of JR-441 for treatment of Sanf..:

Muschol, Nicole M. ; Lindschau, Mona ; Quitmann, Christina...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107973 , 2024

Link: https://doi.org/10.1016/..

6

Efficacy and safety of GH treatment in Japanese children wi..:

Ogata, Tsutomu ; Fukami, Maki ; Tanizawa, Kazunori...
Clinical Pediatric Endocrinology. 33 (2024) 2 - p. 43-49 , 2024

Link: https://doi.org/10.1297/..

7

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/..

8

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/..

9

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/..

10

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/..

11

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/..

12

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/..

13

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/..

14

Drug delivery for neuronopathic lysosomal storage diseases:..:

Sato, Yuji ; Minami, Kohtaro ; Hirato, Toru...
Metabolic Brain Disease. 37 (2022) 6 - p. 1745-1756 , 2022

Link: https://doi.org/10.1007/..

15

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/..