Long-term effectiveness of eliglustat treatment: A real-world analysis from the International Collaborative Gaucher Group Gaucher Registry.

Autor: Mistry PK; Yale University School of Medicine, New Haven, Connecticut, USA., Balwani M; Icahn School of Medicine at Mount Sinai, New York, New York, USA., Charrow J; Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, USA., Lorber J; Cedars-Sinai Medical Center, Los Angeles, California, USA., Niederau C; Katholisches Klinikum Oberhausen, Oberhausen, Germany., Carwile JL; Sanofi, Cambridge, Massachusetts, USA., Oliveira-Dos-Santos A; Sanofi, Sao Paolo, Brazil., Perichon MG; Sanofi, Cambridge, Massachusetts, USA., Uslu Cil S; Sanofi, Cambridge, Massachusetts, USA., Kishnani PS; Duke University Medical Center, Durham, North Carolina, USA.

Publikováno v: American journal of hematology [Am J Hematol] 2024 Aug; Vol. 99 (8), pp. 1500-1510. Date of Electronic Publication: 2024 Apr 30.