Current Status and Challenges of Guarantee for Rare Disease Drugs
ZUO Wei, ZHANG Bo, ZHANG Shuyang
2024, 3(2): 155-163. DOI: 10.12376/j.issn.2097-0501.2024.02.001
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Preliminary Exploration on the Establishment of the Department of Rare Diseases at Peking Union Medical College Hospital
SHEN Min, ZHANG Shuyang
2024, 3(2): 164-167. DOI: 10.12376/j.issn.2097-0501.2024.02.002
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Consideration on " Patient-Centered" Drug Development for Rare Diseases
TANG Ling, AI Xing, ZHAO Tian, YANG Zhimin
2024, 3(2): 168-174. DOI: 10.12376/j.issn.2097-0501.2024.02.003
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Application of Decentralized Clinical Trials in the Research and Development of Drugs for Rare Diseases
CUI Huanhuan, TANG Ling, CUI Can, YAO Zhuxing, YANG Zhimin, WANG Haixue
2024, 3(2): 175-180. DOI: 10.12376/j.issn.2097-0501.2024.02.004
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Elements for Ethic Review of Clinical Trials for Rare Disease Drugs
BAI Hua
2024, 3(2): 181-186. DOI: 10.12376/j.issn.2097-0501.2024.02.005
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Advances in the Diagnosis and Treatment of Cushing′s Syndrome
LIU Cen, ZHAO Zinan, CHEN Di, HU Xin
2024, 3(2): 187-194. DOI: 10.12376/j.issn.2097-0501.2024.02.006
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Study on Drug List and Accessibility of Rare Diseases Based on the China′s Second List of Rare Diseases
LIU Qingyang, LIU Xin, ZUO Wei, WANG Shaohong, ZHANG Bo, ZHANG Shuyang
2024, 3(2): 195-201. DOI: 10.12376/j.issn.2097-0501.2024.02.007
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Overview and Analysis of Rare Disease Drugs Approved for Market in China, the United States, the European Union, and Japan in 2023
WANG Shaohong, LIU Xin, LIU Qingyang, TANG Yan, ZHANG Bo
2024, 3(2): 202-213. DOI: 10.12376/j.issn.2097-0501.2024.02.008
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Literature Review of Using Bisphosphonates in the Treatment of Calciphylaxis
LIANG Chunsu, WANG Shaohong, ZHANG Yizhou, LIU Xin, NIU Ziran, SHI Yili, ZHANG Bo
2024, 3(2): 214-220. DOI: 10.12376/j.issn.2097-0501.2024.02.009
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Trends in Rare Disease Drug Development
CHEN Rui, ZHANG Shuyang
2024, 3(2): 221-223. DOI: 10.12376/j.issn.2097-0501.2024.02.010
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Aphasia in Right Hemiparesis and Pain in Left Waist: A Deceiving Case in Vascular Ehlers-Danlos Syndrome
FU Yining, ZHOU Jingmin, LI Feng, HUANG Shangzhi, WANG Yining, HONG Xia, LYU Ke, LIU Yaping, LENG Ling, ZHENG Wenjie, QIU Zhengqing, ZHU Yicheng, ZHENG Yuehong, CHEN Yuexin
2024, 3(2): 224-231. DOI: 10.12376/j.issn.2097-0501.2024.02.011
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A Case Report of Myotonic Dystrophy Type 1 Presenting as Acute Respiratory Failure
WANG Yiqi, LIN Jie, XI Jianying, LUO Sushan, ZHENG Yanmei, ZHAO Chongbo
2024, 3(2): 232-236. DOI: 10.12376/j.issn.2097-0501.2024.02.012
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A Case Report of Marfan Syndrome with Pituitary Tumor Which Could be Misdiagnosed as Gigantism
CHEN Yu, LI Jianwei, TAN Huiwen, YU Yerong
2024, 3(2): 237-240. DOI: 10.12376/j.issn.2097-0501.2024.02.013
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A Case Report of Primary Hypertrophic Osteoarthropathy
ZHAO Zongxuan, SUN Liying, CHEN Jia, WANG Yanyuan, CHEN Dan, ZUO Qingyao, DENG Wei, TIAN Wen
2024, 3(2): 241-245. DOI: 10.12376/j.issn.2097-0501.2024.02.014
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Advances in Disease-Modifying Therapy of Neuromyelitis Optic Spectrum Disorders
ZHANG Xuanling, CHEN Chaoyang, WEI Ran, ZHOU Ying
2024, 3(2): 246-251. DOI: 10.12376/j.issn.2097-0501.2024.02.015
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Advances in the Adjunctive Treatment of Lennox-Gastaut Syndrome with Clobazam and Cannabidiol
SHI Jingtian, CHEN Chaoyang, YANG Ting, WEI Ran, ZHANG Xuanling, WANG Zining, HU Xiaojuan, ZHOU Ying
2024, 3(2): 252-259. DOI: 10.12376/j.issn.2097-0501.2024.02.016
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Progress in the Diagnosis and Treatment of Infantile Epileptic Spasm Syndrome
ZHENG Lu, YANG Ting, CHEN Chaoyang, WEI Ran, ZHANG Xuanling, MA Yijie, ZHOU Ying
2024, 3(2): 260-268. DOI: 10.12376/j.issn.2097-0501.2024.02.017
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Thoughts on Selection of Rare Diseases and Prioritized Research Topics
LI Kexin, CHEN Jingdan, ZHANG Dingding, GUO Wudong, ZHENG Jiayin, LI Linkang, ZHAO Kun, ZHANG Shuyang
2024, 3(2): 269-274. DOI: 10.12376/j.issn.2097-0501.2024.02.018
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