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


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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