OVERCOMING MARKET ACCESS AND COMMERCIALIZATION CHALLENGES IN GENE THERAPIES FOR RARE CNS DISEASES: GENE THERAPY CLINICAL TRIALS FOR RARE CNS DISEASES LANDSCAPE

被引:0
|
作者
Dabbous, M. [1 ]
Francois, C. [1 ]
Chachoua, L. [2 ]
Hanna, E. [3 ]
Toumi, M. [1 ]
机构
[1] Aix Marseille Univ, Marseille, France
[2] Aix Marseille Univ, Ivry, France
[3] Creativ Ceut, Paris, France
关键词
D O I
暂无
中图分类号
F [经济];
学科分类号
02 ;
摘要
PND5
引用
收藏
页码:S737 / S738
页数:2
相关论文
共 50 条
  • [1] Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement
    Sevin, Caroline
    Deiva, Kumaran
    FRONTIERS IN MOLECULAR BIOSCIENCES, 2021, 8
  • [2] Access to gene therapy for rare diseases when commercialization is not fit for purpose
    Fox, Thomas
    Bueren, Juan
    Candotti, Fabio
    Fischer, Alain
    Aiuti, Alessandro
    Lankester, Arjan
    AGORA Initiative, Michael
    Albert, Michael
    Bernardo, Maria Ester
    Cavazzana, Marina
    Ehl, Stephan
    Neven, Benedicte
    Prevot, Johan
    Schambach, Axel
    Sevilla, Julian
    Thrasher, Adrian
    Wynn, Rob
    Booth, Claire
    NATURE MEDICINE, 2023, 29 (03) : 518 - 519
  • [3] Access to gene therapy for rare diseases when commercialization is not fit for purpose
    Thomas Fox
    Juan Bueren
    Fabio Candotti
    Alain Fischer
    Alessandro Aiuti
    Arjan Lankester
    Claire Booth
    Nature Medicine, 2023, 29 : 518 - 519
  • [4] Gene Therapies for Rare Diseases
    不详
    JOURNAL OF NUCLEAR MEDICINE, 2022, 63 (01) : 22N - 22N
  • [5] Improving access to gene therapy for rare diseases
    Fox, Thomas A.
    Booth, Claire
    DISEASE MODELS & MECHANISMS, 2024, 17 (06)
  • [6] CNS diseases amenable to gene therapy
    Federoff, HJ
    HUMAN GENE THERAPY: CURRENT OPPORTUNITIES AND FUTURE TRENDS, 2003, 43 : 117 - 158
  • [7] Ensuring patient access to gene therapies for rare diseases: Navigating reimbursement and coverage challenges
    Berry, Diane
    Hickey, Carolyn
    Kahlman, Lisa
    Long, James
    Markus, Christina
    Mccombs, Caitlin K.
    MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2025, 33 (01)
  • [8] Gene therapies for rare neurological diseases
    Burgunder, J.
    EUROPEAN JOURNAL OF NEUROLOGY, 2022, 29 : 41 - 41
  • [9] Curative gene therapies for rare diseases
    Rocio Maldonado
    Sami Jalil
    Kirmo Wartiovaara
    Journal of Community Genetics, 2021, 12 : 267 - 276
  • [10] Curative gene therapies for rare diseases
    Maldonado, Rocio
    Jalil, Sami
    Wartiovaara, Kirmo
    JOURNAL OF COMMUNITY GENETICS, 2021, 12 (02) : 267 - 276