Long‐term effectiveness of eliglustat treatment: A real‐world analysis from the International Collaborative Gaucher Group Gaucher Registry
- Published on 07/05/2024
- Reading time: 5 min.
Pramod K. Mistry 1, Manisha Balwani 2, Joel Charrow 3, Jeremy Lorber 4, Claus Niederau 5, Jenny L. Carwile 6, Antonio Oliveira‐dos‐Santos 7, Maria Gabriela Perichon 6, Sefika Uslu Cil 6, Priya S. Kishnani 8
1
Yale University School of Medicine
New Haven
Connecticut
USA
2
Icahn School of Medicine at Mount Sinai
New York
New York
USA
3
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago
Illinois
USA
4
Cedars‐Sinai Medical Center
Los Angeles
California
USA
5
Katholisches Klinikum Oberhausen
Oberhausen
Germany
6
Sanofi
Cambridge
Massachusetts
USA
7
Sanofi
Sao Paolo
Brazil
8
Duke University Medical Center
Durham
North Carolina
USA
Abstract
Gaucher disease type 1 (GD1) is known for phenotypic heterogeneity and varied natural history. Registrational clinical trials enrolled narrowly defined phenotypes, but greater diversity is encountered in clinical practice. We report real‐world outcomes with long‐term eliglustat treatment in adults with GD1 in the International Collaborative Gaucher Group Gaucher Registry. Among 5985 GD1 patients in the Registry as of January 6, 2023, 872 started eliglustat at ≥18 years...
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