Drug reduces excess iron in ultra-rare neurodegenerative disease

被引:0
|
作者
Sarah Lemprière
机构
[1] Nature Reviews Neurology,
来源
Nature Reviews Neurology | 2019年 / 15卷
关键词
D O I
暂无
中图分类号
学科分类号
摘要
引用
收藏
页码:492 / 493
页数:1
相关论文
共 50 条
  • [21] An Ultra-rare Disease? Where Do We Go from Here?
    Irvine, Glenn
    Irvine, Ginger
    TREMOR AND OTHER HYPERKINETIC MOVEMENTS, 2013, 3
  • [22] The NINDS Ultra-Rare Gene-based Therapy Network: an URGenT Need for Ultra-rare Neurological Diseases
    Broome, Ann-Marie
    Schor, Nina
    Tamiz, Amir
    Morris, Jill
    Boshoff, Chris
    Skiadopoulos, Mario
    Bachman, Julia
    Koroshetz, Walter
    NEUROTHERAPEUTICS, 2021, 18 (03) : 2138 - 2138
  • [23] Drug repurposing and phenotypic screening: innovative strategies for treating ultra-rare disorders
    Paquot, Adrien
    Deprez, Benoit
    Beghyn, Terence
    FRONTIERS IN MEDICINE, 2024, 11
  • [24] 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
  • [25] Are Costs Decisive in the Incorporation of Medicines for the Treatment of Ultra-Rare Diseases in Brazil? Trends in Healthcare Coverage for Ultra-Rare Diseases
    Lopes, Luciane Cruz
    Pereira Curado, Daniel da Silva
    Machado-Rugolo, Juliana
    de Paula de Sordi, Monica Aparecida
    Komoda, Denis Satoshi
    Modelli de Andrade, Luis Gustavo
    Theresa Weber, Silke Anna
    Tarride, Jean Eric
    Thabane, Lehana
    Santos, Marisa
    de Almeida Cardoso, Marilia Mastrocolla
    PHARMACOEPIDEMIOLOGY AND DRUG SAFETY, 2024, 33 : 673 - 674
  • [26] A call to arms against ultra-rare diseases
    Crooke, Stanley T.
    NATURE BIOTECHNOLOGY, 2021, 39 (06) : 671 - 677
  • [27] Reimbursement of drugs for ultra-rare diseases in Lithuania
    Karinauske, Egle
    Lasys, Tomas
    Kasciuskeviciute, Skaiste
    Usaite, Akvile
    Stankeviciute, Simona
    Sadauskas, Vladas
    EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY, 2021, 77 (SUPPL 1) : 32 - 32
  • [28] Ultra-rare ultra-care: Assessing the impact of caring for children with ultra rare diseases
    Domaradzki, Jan
    Walkowiak, Dariusz
    EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY, 2024, 48 : 78 - 84
  • [29] TOO ULTRA-RARE FOR CARE? ORPHAN DRUG AVAILABILITY FOR RESPIRATORY DISEASES: A SYSTEMATIC REVIEW
    Matthews, H.
    Lovelace, M.
    Freitag, A.
    Iheanacho, I.
    VALUE IN HEALTH, 2019, 22 : S857 - S857
  • [30] THE ROLE OF ULTRA-RARE CODING VARIANTS IN ADHD
    Demontis, Ditte
    Satterstrom, Kyle
    Duan, Jinjie
    Lescai, Francesco
    Ostergaard, Soren Dinesen
    Lesch, Klaus-Peter
    Werge, Thomas
    Mortensen, Preben Bo
    Glerup, Simon
    Franke, Barbara
    Hougaard, David M.
    Reif, Andreas
    Daly, Mark
    Neale, Benjamin
    Borglum, Anders D.
    EUROPEAN NEUROPSYCHOPHARMACOLOGY, 2019, 29 : S724 - S725