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Future Rare Diseases Volume 4, 2023 - Issue 1

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Plain language summary of a study looking at the long-term benefits of enzyme replacement therapy in children and teenagers with Gaucher disease type 3

Amal El-Beshlawy1 Pediatric Hospital of Cairo University, Cairo, Egypt

Anna Tylki-Szymanska2 Children's Memorial Health Institute, Warsaw, Poland

Nadia Belmatoug3 Referral Center for Lysosomal Diseases, University Hospital Paris Nord-Val de Seine Assistance Publique-Hôpitaux de Paris, France

Pramod K Mistry4 Yale University School of Medicine, New Haven, CT, USA

Article: FRD52 | Received 14 Jul 2023, Accepted 16 Nov 2023, Published online: 06 Feb 2024

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https://doi.org/10.2217/frd-2023-0015
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