ECONOMIC MODEL FOR A GENE THERAPY IN AN ULTRA-RARE DISEASE: EXPERT VALIDATION OF APPROACH AND ASSUMPTIONS

被引：0
|
作者：
Sopena, L. [1 ]
Simons, C. [2 ]
Mumford, A. [3 ]
Bennison, C. [4 ]
Irvin, W. [1 ]
Paris, J. J. [5 ]
Buesch, K. [6 ]
机构：
[1] Initiate Consultancy, London, Lon, England
[2] OPEN Hlth Grp, York, Nyk, England
[3] Initiate Consultancy, Northampton, England
[4] OPEN Hlth, York, N Yorkshire, England
[5] OPEN Hlth, Oxford, England
[6] PTC Therapeut Switzerland GmbH, Steinhausen, Switzerland
来源：
VALUE IN HEALTH | 2022年 / 25卷 / 01期
关键词：
D O I：
暂无
中图分类号：
F [经济];
学科分类号：
02 ;
摘要：
POSB96
引用
收藏
页码：S79 / S79
页数：1
相关论文
共 50 条
  • [21] Integrated Applied Clinical Pharmacology in the Advancement of Rare and Ultra-Rare Disease Therapeutics
    Ryder, Steven
    CLINICAL PHARMACOLOGY & THERAPEUTICS, 2024, 116 (06) : 1485 - 1495
  • [22] Drug reduces excess iron in ultra-rare neurodegenerative disease
    Lempriere, Sarah
    NATURE REVIEWS NEUROLOGY, 2019, 15 (09) : 492 - 493
  • [23] Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery
    Wood, Jill
    Sames, Lori
    Moore, Allison
    Ekins, Sean
    DRUG DISCOVERY TODAY, 2013, 18 (21-22) : 1043 - 1051
  • [24] Zebrafish models ultra-rare X-linked disease
    Linda Koch
    Nature Reviews Genetics, 2014, 15 (12) : 778 - 778
  • [25] Child with ultra-rare disease gets a treatment just for her
    Nature, 2024, 632 (8026) : 711 - 711
  • [26] Triheptanoin-Novel therapeutic approach for the ultra-rare disease mitochondrial malate dehydrogenase deficiency
    Laemmle, Alexander
    Steck, Andrea Lisa
    Schaller, Andre
    Kurth, Sandra
    Hoigne, Eveline Perret
    Felser, Andrea Deborah
    Slavova, Nedelina
    Salvisberg, Claudia
    Atencio, Mariana
    Mochel, Fanny
    Nuoffer, Jean-Marc
    Gautschi, Matthias
    MOLECULAR GENETICS AND METABOLISM REPORTS, 2021, 29
  • [27] Relocation of study participants for rare and ultra-rare disease trials: Ethics and operations
    Gelinas, Luke
    Crawford, Brian
    Kelman, Ariella
    Bierer, Barbara E.
    CONTEMPORARY CLINICAL TRIALS, 2019, 84
  • [28] Why we should care about ultra-rare disease
    Harari, Sergio
    EUROPEAN RESPIRATORY REVIEW, 2016, 25 (140): : 101 - 103
  • [29] An Ultra-rare Disease? Where Do We Go from Here?
    Irvine, Glenn
    Irvine, Ginger
    TREMOR AND OTHER HYPERKINETIC MOVEMENTS, 2013, 3
  • [30] Mapping of resources for undiagnosed and newly diagnosed ultra-rare disease networks
    Gumus, G. H.
    Broley, S.
    Patch, C.
    del Olmo, M. Montolio
    Boycott, K.
    Faivre, L.
    Zurek, B.
    Macek, M.
    Graessner, H.
    Bellagambi, S.
    Stemkens, D.
    van Doorne, C.
    Metcalfe, A.
    Costa, A.
    Robert, G.
    Roberts, L.
    Pritchard, M.
    Scheffer, H.
    Boulanger, V.
    Lewis, J.
    Cederroth, H.
    Cederroth, M.
    Arias, P.
    Bros-Facer, V.
    EUROPEAN JOURNAL OF HUMAN GENETICS, 2020, 28 (SUPPL 1) : 783 - 784
12345