Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys

Authors

Derralynn Hughes (ORCID: https://orcid.org/0000-0003-4531-9173)
Shoshana Revel‐Vilk (ORCID: https://orcid.org/0000-0001-9151-0337)
Uma Ramaswami (ORCID: https://orcid.org/0000-0002-4703-7447)
Guillem Pintos‐Morell (ORCID: https://orcid.org/0000-0002-9347-2386)
Aidan Gill
Kathy Nicholls (ORCID: https://orcid.org/0000-0002-1297-8722)
Majdolen Istaiti (ORCID: https://orcid.org/0000-0001-7598-2828)
Özlem Göker-Alpan (ORCID: https://orcid.org/0000-0003-4758-6793)
Jaco Botha (ORCID: https://orcid.org/0000-0002-8889-9959)
Ricardo Reisin (ORCID: https://orcid.org/0000-0002-7278-4639)
Roberto Giugliani (ORCID: https://orcid.org/0000-0001-9655-3686)
Maurizio Scarpa

Institutions

University of Udine (IT)
Universidade Federal do Rio Grande do Sul (BR)
The Royal Melbourne Hospital (AU)
The University of Melbourne (AU)
Royal Free London NHS Foundation Trust (GB)
Hebrew University of Jerusalem (IL)
Santa Casa Hospital (BR)
Shaare Zedek Medical Center (IL)
Takeda (Switzerland) (CH)
Vall d'Hebron Institut de Recerca (ES)
Lysosomal and Rare Disorders Research and Treatment Center (US)
National Institute on Population Medical Genetics (BR)
Takeda (United States) (US)
Hospital Británico de Buenos Aires (AR)
University College London (GB)

Publication Details

Journal: Orphanet Journal of Rare Diseases
Published: 2025-03-28
DOI: https://doi.org/10.1186/s13023-024-03416-2
Citations: 2
Primary Topic: Lysosomal Storage Disorders Research
Type: article
Field-Weighted Citation Impact: 2.33

Funders

Takeda Pharmaceuticals International

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article

Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys

Derralynn Hughes, Shoshana Revel‐Vilk, Uma Ramaswami, Guillem Pintos‐Morell et al.

Mar 28, 2025

2 citations

Orphanet Journal of Rare Diseases

Lysosomal Storage Disorders Research

2.33

article

Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys

Derralynn Hughes, Shoshana Revel‐Vilk, Uma Ramaswami, Guillem Pintos‐Morell, Aidan Gill, Kathy Nicholls, Majdolen Istaiti, Özlem Göker-Alpan, Jaco Botha, Ricardo Reisin, Roberto Giugliani, Maurizio Scarpa

March 28, 2025article en

2 citations

Abstract

No abstract available for this paper.

Orphanet Journal of Rare DiseasesVol. 20(1)

University of Udine (IT), Universidade Federal do Rio Grande do Sul (BR), The Royal Melbourne Hospital (AU), The University of Melbourne (AU), Royal Free London NHS Foundation Trust (GB), Hebrew University of Jerusalem (IL), Santa Casa Hospital (BR), Shaare Zedek Medical Center (IL), Takeda (Switzerland) (CH), Vall d'Hebron Institut de Recerca (ES), Lysosomal and Rare Disorders Research and Treatment Center (US), National Institute on Population Medical Genetics (BR), Takeda (United States) (US), Hospital Británico de Buenos Aires (AR), University College London (GB)

Takeda Pharmaceuticals International

Openalex Percentile: Top 13%

Lysosomal Storage Disorders Research

2.33

DOI Open Access PDF OpenAlex

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Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys — Derralynn Hughes, Shoshana Revel‐Vilk, et al. · Orphanet Journal of Rare Diseases (2025) | TGRS Research Map | TGRS