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A 10-year follow-up of high-dose ambroxol treatment combined with enzyme replacement therapy for neuropathic Gaucher disease.
Hwang, Soojin; Bae, Hyunwoo; Yoon, Ji-Hee; Kim, Dohyung; Do, Hyo-Sang; Heo, Sun Hee; Kim, Soyoung; Yoo, Han-Wook; Istaiti, Majdolen; Zimran, Ari; Lee, Beom Hee.
Affiliation
  • Hwang S; Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Republic of Korea.
  • Bae H; Medical Genetics Center, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Republic of Korea.
  • Yoon JH; Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Republic of Korea.
  • Kim D; Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Republic of Korea.
  • Do HS; Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Republic of Korea.
  • Heo SH; Asan Institute for Life Sciences, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
  • Kim S; Asan Institute for Life Sciences, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
  • Yoo HW; Asan Institute for Life Sciences, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
  • Istaiti M; Department of Pediatrics, CHA Bundang Medical Center, CHA University, Seongnam, Republic of Korea.
  • Zimran A; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem, Israel.
  • Lee BH; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem, Israel.
Am J Hematol ; 99(7): 1396-1399, 2024 07.
Article in En | MEDLINE | ID: mdl-38562044
ABSTRACT
High-dose ambroxol therapy combined with ERT in patients with neuropathic Gaucher disease.
Subject(s)
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Enzyme Replacement Therapy / Ambroxol / Gaucher Disease / Glucosylceramidase Limits: Adult / Female / Humans / Male / Middle aged Language: En Journal: Am J Hematol Year: 2024 Document type: Article Country of publication: United States
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Enzyme Replacement Therapy / Ambroxol / Gaucher Disease / Glucosylceramidase Limits: Adult / Female / Humans / Male / Middle aged Language: En Journal: Am J Hematol Year: 2024 Document type: Article Country of publication: United States