Advancing orphan drug development for rare diseases

被引:0
|
作者
Ko, Jung Min [1 ,2 ]
机构
[1] Seoul Natl Univ, Coll Med, Dept Pediat, Div Clin Genet, 103 Daehak Ro, Seoul 03080, South Korea
[2] Seoul Natl Univ Hosp, Rare Dis Ctr, Seoul, South Korea
关键词
D O I
10.3345/cep.2023.01109
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
引用
收藏
页码:356 / 357
页数:2
相关论文
共 50 条
  • [1] RARE DISEASES, DRUG DEVELOPMENT, AND AIDS - THE IMPACT OF THE ORPHAN DRUG-ACT
    ARNO, PS
    BONUCK, K
    DAVIS, M
    MILBANK QUARTERLY, 1995, 73 (02): : 231 - 252
  • [2] DRUG DEVELOPMENT FOR ORPHAN DISEASES
    CERAMI, A
    AMERICAN JOURNAL OF MEDICINE, 1978, 65 (03): : 407 - 408
  • [3] Development of orphan drugs for rare diseases
    Yoo, Han-Wook
    CLINICAL AND EXPERIMENTAL PEDIATRICS, 2024, 67 (07) : 315 - 327
  • [4] Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases
    Micallef, Joelle
    Blin, Olivier
    THERAPIE, 2020, 75 (02): : 133 - 139
  • [5] Advancing drug development in neuro-orphan indications
    Trad, M.
    Boyer, S.
    VanBelle, C.
    Hughes, L.
    Moody, B.
    JOURNAL OF THE NEUROLOGICAL SCIENCES, 2017, 381 : 967 - 968
  • [6] The impact of orphan drug policies in treating rare diseases
    Weerasooriya, Shalini U.
    HEALTH INFORMATION AND LIBRARIES JOURNAL, 2019, 36 (02): : 179 - 184
  • [7] The Orphan Drug Act: Restoring the Mission to Rare Diseases
    Daniel, Michael G.
    Pawlik, Timothy M.
    Fader, Amanda N.
    Esnaola, Nestor F.
    Makary, Martin A.
    AMERICAN JOURNAL OF CLINICAL ONCOLOGY-CANCER CLINICAL TRIALS, 2016, 39 (02): : 210 - 213
  • [8] A Personal Perspective of Orphan Drug Development for Rare Diseases: A Golden Opportunity or an Unsustainable Future?
    Oo, Charles
    Rusch, Lorraine M.
    JOURNAL OF CLINICAL PHARMACOLOGY, 2016, 56 (03): : 257 - 259
  • [9] The Orphan Drug Act and the Development of Stem Cell-Based Products for Rare Diseases
    Freeman, Scott N.
    Burke, Kathryn A.
    Imoisili, Menfo A.
    Cote, Timothy R.
    CELL STEM CELL, 2010, 7 (03) : 283 - 287
  • [10] Rare lung disease and orphan drug development
    Spagnolo, Paolo
    du Bois, Roland M.
    Cottin, Vincent
    LANCET RESPIRATORY MEDICINE, 2013, 1 (06): : 479 - 487