Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). <i>J. Clin. Med.</i> 2022, <i>11</i>, 5158".

Hughes, Derralynn A; Deegan, Patrick; Giraldo, Pilar; Göker-Alpan, Özlem; Lau, Heather; Lukina, Elena; Revel-Vilk, Shoshana; Scarpa, Maurizio; Botha, Jaco; Gadir, Noga; Zimran, Ari

Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158".

Hughes, Derralynn A; Deegan, Patrick; Giraldo, Pilar; Göker-Alpan, Özlem; Lau, Heather; Lukina, Elena; Revel-Vilk, Shoshana; Scarpa, Maurizio; Botha, Jaco; Gadir, Noga; Zimran, Ari.

Afiliación

Hughes DA; LSD Unit, Royal Free London NHS Foundation Trust, University College London, London NW3 2QG, UK.
Deegan P; Addenbrookes Hospital, Cambridge CB2 0QQ, UK.
Giraldo P; CIBER de Enfermedades Raras, IIS Aragon, 50009 Zaragoza, Spain.
Göker-Alpan Ö; Translational Research Unit, IIS Aragon, 50009 Zaragoza, Spain.
Lau H; Lysosomal Disorders Unit and Center for Clinical Trials, O&O Alpan LLC, Fairfax, VA 22030, USA.
Lukina E; Department of Neurology, New York University School of Medicine, New York, NY 10016, USA.
Revel-Vilk S; Department of Orphan Diseases, National Research Center for Hematology, 125167 Moscow, Russia.
Scarpa M; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
Botha J; The Faculty of Medicine, Hebrew University, Jerusalem 9112102, Israel.
Gadir N; Centre for Rare Diseases, Academic Medical Centre Hospital of Udine, 33100 Udine, Italy.
Zimran A; Takeda Pharmaceuticals International AG, 8152 Zurich, Switzerland.

J Clin Med ; 12(12)2023 Jun 13.

Article en En | MEDLINE | ID: mdl-37373710

ABSTRACT

ABSTRACT

Thank you for allowing us the opportunity to respond to the commentary from Mistry and colleagues (

Comment:

The two substrate reduction therapies for type 1 Gaucher disease are not equivalent) [...].

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: J Clin Med Año: 2023 Tipo del documento: Article País de afiliación: Reino Unido

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