Gene-Targeted Therapies and Palliative Care in Children with Spinal Muscular Atrophy Type I: No Intrinsic Contradiction

被引：0
|
作者：
von der Hagen, Maja [1 ]
Nolte-Buchholtz, Silke [2 ]
Janisch, Maria [2 ]
Pietz, Joachim [3 ]
Blankenburg, Markus [4 ,5 ]
Ziegler, Andreas [6 ]
机构：
[1] Tech Univ Dresden, Abt Neuropadiat, Med Fak Carl Gustav Carus, Fetscherstr 74, D-01307 Dresden, Germany
[2] Tech Univ Dresden, Sachs Kinderpalliat Zentrum, Med Fak Carl Gustav Carus, Klin Kinder & Jugendmed, Dresden, Germany
[3] Palliat Care Team Children & Adolescents, Frankfurt, Germany
[4] Univ Witten Herdecke, Zentrum Kinder Jugend & Frauenmed, Padiat Neurol Psychosomat & Schmerztherapie, Klinikum Stuttgart Olgahosp, Witten, Germany
[5] Univ Witten Herdecke, Fak Gesundheit, Witten, Germany
[6] Univ Klinikum Heidelberg, Zentrum Kinder & Jugendmed Heidelberg, Sekt Neuropadiat & Stoffwechselmed, Heidelberg, Germany
来源：
JOURNAL OF PALLIATIVE MEDICINE | 2021年 / 24卷 / 02期
关键词：
D O I：
10.1089/jpm.2020.0505
中图分类号：
R19 [保健组织与事业（卫生事业管理）];
学科分类号：
摘要：
引用
收藏
页码：162 / 163
页数：2
相关论文
共 50 条
  • [1] Palliative care in children with spinal muscular atrophy type I: What do they need?
    Garcia-Salido, Alberto
    Garcia de Paso-Mora, Maria
    Monleon-Luque, Manuel
    Martino-Alba, Ricardo
    PALLIATIVE & SUPPORTIVE CARE, 2015, 13 (02) : 313 - 317
  • [2] Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain
    Sumner, Charlotte J.
    Crawford, Thomas O.
    JOURNAL OF CLINICAL INVESTIGATION, 2018, 128 (08): : 3219 - 3227
  • [3] Which care for children with spinal muscular atrophy type I and their families at hospital?
    Jouinot, Patricia
    Estournet, Brigitte
    MEDECINE PALLIATIVE, 2010, 9 (06): : 303 - 308
  • [4] Current and emerging targeted therapies for spinal muscular atrophy
    Lakhina, Yuliya
    Boulis, Nicholas M.
    Donsante, Anthony
    EXPERT REVIEW OF NEUROTHERAPEUTICS, 2023, 23 (12) : 1189 - 1199
  • [5] The role of palliative care in advanced muscular dystrophy and spinal muscular atrophy
    Parker, D
    Maddocks, I
    Stern, LM
    JOURNAL OF PAEDIATRICS AND CHILD HEALTH, 1999, 35 (03) : 245 - 250
  • [6] The Experience of Families With Children With Spinal Muscular Atrophy Type I Across Health Care Systems
    Murrell, Diane V.
    Lotze, Timothy E.
    Farber, Harold J.
    Crawford, Claire A.
    Wiemann, Constance M.
    JOURNAL OF CHILD NEUROLOGY, 2017, 32 (11) : 917 - 923
  • [7] Genetic therapies for spinal muscular atrophy type 1
    Aartsma-Rus, Annemieke
    LANCET NEUROLOGY, 2018, 17 (02): : 111 - 112
  • [8] Mutation of gene in spinal muscular atrophy respiratory distress type I
    Wong, Virginia C. N.
    Chung, Brian H. Y.
    Li, Susanna
    Goh, Winnie
    Lee, So Lun
    PEDIATRIC NEUROLOGY, 2006, 34 (06) : 474 - 477
  • [9] Predictive energy equations for spinal muscular atrophy type I children
    Bertoli, Simona
    De Amicis, Ramona
    Bedogni, Giorgio
    Foppiani, Andrea
    Leone, Alessandro
    Ravella, Simone
    Mastella, Chiara
    Baranello, Giovanni
    Masson, Riccardo
    Bertini, Enrico
    D'Amico, Adele
    Pedemonte, Marina
    Bruno, Claudio
    Agosto, Caterina
    Giaquinto, Ester
    Bassano, Michela
    Battezzati, Alberto
    AMERICAN JOURNAL OF CLINICAL NUTRITION, 2020, 111 (05): : 983 - 996
  • [10] Matching pairs difficulty in children with spinal muscular atrophy type I
    Polido, Graziela Jorge
    Barbosa, Alessandra Ferreira
    Morimoto, Carlos Hitoshi
    Caromano, Fatima Aparecida
    Favero, Francis Meire
    Zanoteli, Edmar
    Reed, Umbertina Conti
    Voos, Mariana Callil
    NEUROMUSCULAR DISORDERS, 2017, 27 (05) : 419 - 427
12345