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The clinical characteristics and outcomes of patients with systemic sclerosis with myocardial involvement
Author: He, Huilin; Lai, Jinzhi; Zhou, Jiaxin; Hou, Yong; Xu, Dong; Li, Mengtao; Zeng, Xiaofeng
Journal: ORPHANET JOURNAL OF RARE DISEASES. 2023; Vol. 18, Issue 1, pp. -. DOI: 10.1186/s13023-023-02699-1
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Association between one-session bilateral whole-lung lavage and periprocedural complications in patients with pulmonary alveolar proteinosis: a retrospective cohort study
Author: Xu, Xiaohan; Qiu, Huanrong; Chen, Fei; Zhang, Yuelun; Tian, Xinlun; Liu, Yongjian; Wang, Ping; Zhu, Bo; Huang, Yuguang
Journal: ORPHANET JOURNAL OF RARE DISEASES. 2023; Vol. 18, Issue 1, pp. -. DOI: 10.1186/s13023-023-02691-9
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The global prevalence and ethnic heterogeneity of iron-refractory iron deficiency anaemia
Author: Fan, Shanghua; Zhao, Ting; Sun, Liu
Journal: ORPHANET JOURNAL OF RARE DISEASES. 2023; Vol. 18, Issue 1, pp. -. DOI: 10.1186/s13023-022-02612-2
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A patient advocating for transparent science in rare disease research
Author: Yang, Richard Rui
Journal: ORPHANET JOURNAL OF RARE DISEASES. 2023; Vol. 18, Issue 1, pp. -. DOI: 10.1186/s13023-022-02557-6
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Prevalence of depression and anxiety, and their relationship to social support among patients and family caregivers of rare bone diseases
Author: Lai, Xuefeng; Jiang, Yuling; Sun, Yue; Zhang, Zhijun; Wang, Shengfeng
Journal: ORPHANET JOURNAL OF RARE DISEASES. 2023; Vol. 18, Issue 1, pp. -. DOI: 10.1186/s13023-022-02611-3
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Glutamate dehydrogenase hyperinsulinism: mechanisms, diagnosis, and treatment
Author: Zeng, Qiao; Sang, Yan-Mei
Journal: ORPHANET JOURNAL OF RARE DISEASES. 2023; Vol. 18, Issue 1, pp. -. DOI: 10.1186/s13023-023-02624-6
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Effects of early recombinant human growth hormone treatment in young Chinese children with Prader-Willi syndrome
Author: Gao, Ying; Yang, Li-Li; Dai, Yang-Li; Shen, Zheng; Zhou, Qiong; Zou, Chao-Chun
Journal: ORPHANET JOURNAL OF RARE DISEASES. 2023; Vol. 18, Issue 1, pp. -. DOI: 10.1186/s13023-023-02615-7
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Ultrasound-guided interlaminar approach for nusinersen administration in patients with spinal muscular atrophy with spinal fusion or severe scoliosis
Author: Wei, Cuijie; Liang, Zhenwei; Wu, Ying; Liu, Shan; Qiu, Jianxing; Meng, Lingchao; Li, Chunde; Li, Shuang; Bao, Xinhua; Wang, Zhaoxia; Chen, Luzeng; Xiong, Hui
Journal: ORPHANET JOURNAL OF RARE DISEASES. 2023; Vol. 18, Issue 1, pp. -. DOI: 10.1186/s13023-023-02630-8
