Efficacy and safety of GH treatment in Japanese children with short stature due to <i>SHOX</i> deficiency: a randomized phase 3 study-Reference-Cited by-同舟云学术

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Efficacy and safety of GH treatment in Japanese children with short stature due to <i>SHOX</i> deficiency: a randomized phase 3 study

Published:2024 Issue:2 Volume:33 Page:43-49
ISSN:0918-5739
Container-title:Clinical Pediatric Endocrinology
language:en
Short-container-title:Clin Pediatr Endocrinol

Author:

Ogata Tsutomu¹²,Fukami Maki³,Tanizawa Kazunori⁴,Yamamoto Tatsuyoshi⁴,Sato Yuji⁴,Hirai Hideaki⁴,Takasao Naoko⁴,Ibaraki Ryo⁴,Noda Marin⁴

Affiliation:

1. Department of Pediatrics and Biochemistry, Hamamatsu University School of Medicine, Hamamatsu, Japan

2. Department of Pediatrics, Hamamatsu Medical Center, Hamamatsu, Japan

3. Department of Molecular Endocrinology, National Research Institute for Child Health and Development, Tokyo, Japan

4. Development Division, JCR Pharmaceuticals, Ashiya, Japan

Publisher

Japanese Society for Pediatric Endocrinology

Link

https://www.jstage.jst.go.jp/article/cpe/33/2/33_2023-0070/_pdf

Reference16 articles.

1. 1. Rao, E, Weiss, B, Fukami, M, Rump, A, Niesler, B, Mertz, A, et al. Pseudoautosomal deletions encompassing a novel homeobox gene cause growth failure in idiopathic short stature and Turner syndrome. Nat Genet 1997;16: 54–63.

2. 2. Marchini, A, Ogata, T, Rappold, GA. A track record on SHOX: from basic research to complex models and therapy. Endocr Rev 2016;37: 417–48.

3. 3. Fukami, M, Shindo, J, Ogata, T, Kageyama, I, Kamimaki, T. SHOX far-downstream deletion in a patient with nonsyndromic short stature. Am J Med Genet A 2022;188: 2173–7.

4. 4. Ross, MT, Grafham, DV, Coffey, AJ, Scherer, S, McLay, K, Muzny, D, et al. The DNA sequence of the human X chromosome. Nature 2005;434: 325–37.

5. 5. Fukami, M, Seki, A, Ogata, T. SHOX haploinsufficiency as a cause of syndromic and nonsyndromic short stature. Mol Syndromol 2016;7: 3–11.

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