Longitudinal natural history of type I spinal muscular atrophy: a critical review

被引：0
|
作者：
Eugenio Mercuri
Simona Lucibello
Marco Perulli
Giorgia Coratti
Roberto de Sanctis
Maria Carmela Pera
Marika Pane
Jacqueline Montes
Darryl C. de Vivo
Basil T. Darras
Stephen J. Kolb
Richard S. Finkel
机构：
[1] Catholic University,Paediatric Neurology
[2] Fondazione Policlinico Universitario Agostino Gemelli IRCCS,Centro Clinico Nemo, Policlinico Gemelli
[3] Columbia University Medical Center,Departments of Rehabilitation and Regenerative Medicine
[4] Columbia University Medical Center,Department of Neurology
[5] Boston Children’s Hospital,Department of Neurology
[6] Harvard Medical School,Department of Neurology
[7] The Ohio State University Wexner Medical Center,Department of Biological Chemistry & Pharmacology
[8] The Ohio State University Wexner Medical Center,Nemours Children’s Hospital
[9] University of Central Florida College of Medicine,undefined
来源：
Orphanet Journal of Rare Diseases | / 15卷
关键词：
Spinal muscular atrophy; Natural history; CHOP INTEND;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
引用
收藏
相关论文
共 50 条
  • [1] Longitudinal natural history of type I spinal muscular atrophy: a critical review
    Mercuri, Eugenio
    Lucibello, Simona
    Perulli, Marco
    Coratti, Giorgia
    de Sanctis, Roberto
    Pera, Maria Carmela
    Pane, Marika
    Montes, Jacqueline
    de Vivo, Darryl C.
    Darras, Basil T.
    Kolb, Stephen J.
    Finkel, Richard S.
    ORPHANET JOURNAL OF RARE DISEASES, 2020, 15 (01)
  • [2] Natural history in spinal muscular atrophy Type I in Taiwanese population: A longitudinal study
    Ou, Shan-Fu
    Ho, Che-Sheng
    Lee, Wang-Tso
    Lin, Kuang-Lin
    Jones, Cynthia C.
    Jong, Yuh-Jyh
    BRAIN & DEVELOPMENT, 2021, 43 (01) : 127 - 134
  • [3] THE NATURAL-HISTORY OF TYPE-I (SEVERE) SPINAL MUSCULAR-ATROPHY
    THOMAS, NH
    DUBOWITZ, V
    NEUROMUSCULAR DISORDERS, 1994, 4 (5-6) : 497 - 502
  • [4] Natural history of lung function in spinal muscular atrophy
    Wijngaarde, Camiel A.
    Veldhoen, Esther S.
    van Eijk, Ruben P. A.
    Stam, Marloes
    Otto, Louise A. M.
    Asselman, Fay-Lynn
    Wosten-van Asperen, Roelie M.
    Hulzebos, Erik H. J.
    Verweij-van den Oudenrijn, Laura P.
    Bartels, Bart
    Cuppen, Inge
    Wadman, Renske, I
    van den Berg, Leonard H.
    van der Ent, Cornelis K.
    van der Pol, W. Ludo
    ORPHANET JOURNAL OF RARE DISEASES, 2020, 15 (01)
  • [5] Natural history of lung function in spinal muscular atrophy
    Camiel A. Wijngaarde
    Esther S. Veldhoen
    Ruben P. A. van Eijk
    Marloes Stam
    Louise A. M. Otto
    Fay-Lynn Asselman
    Roelie M. Wösten-van Asperen
    Erik H. J. Hulzebos
    Laura P. Verweij-van den Oudenrijn
    Bart Bartels
    Inge Cuppen
    Renske I. Wadman
    Leonard H. van den Berg
    Cornelis K. van der Ent
    W. Ludo van der Pol
    Orphanet Journal of Rare Diseases, 15
  • [6] History review of spinal muscular atrophy
    Bieniaszewska, Aleksandra
    Gajewska, Ewa
    AKTUALNOSCI NEUROLOGICZNE, 2022, 22 (01): : 32 - 38
  • [7] Developmental milestones in type I spinal muscular atrophy
    De Sanctis, Roberto
    Coratti, Giorgia
    Pasternak, Amy
    Montes, Jacqueline
    Pane, Marika
    Mazzone, Elena S.
    Young, Sally Dunaway
    Salazar, Rachel
    Quigley, Janet
    Pera, Maria C.
    Antonaci, Laura
    Lapenta, Leonardo
    Glanzman, Allan M.
    Tiziano, Danilo
    Muntoni, Francesco
    Darras, Basil T.
    De Vivo, Darryl C.
    Finkel, Richard
    Mercuri, Eugenio
    NEUROMUSCULAR DISORDERS, 2016, 26 (11) : 754 - 759
  • [8] Natural History of Type 1 Spinal Muscular Atrophy in a Series of Argentinian Children
    Aguerre, V
    De Castro, F.
    Mozzoni, J.
    Gravina, L. P.
    Araoz, H., V
    Monges, S.
    JOURNAL OF NEUROMUSCULAR DISEASES, 2020, 7 (04) : 453 - 458
  • [9] Natural history of 10-meter walk/run test performance in spinal muscular atrophy: A longitudinal analysis
    Krosschell, Kristin J.
    Townsend, Elise L.
    Kiefer, Michael
    Simeone, Sarah D.
    Zumpf, Katelyn
    Welty, Leah
    Swoboda, Kathryn J.
    NEUROMUSCULAR DISORDERS, 2022, 32 (02) : 125 - 134
  • [10] Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study
    Claude Cances
    Dmitry Vlodavets
    Giacomo Pietro Comi
    Riccardo Masson
    Maria Mazurkiewicz-Bełdzińska
    Kayoko Saito
    Edmar Zanoteli
    Angela Dodman
    Muna El-Khairi
    Ksenija Gorni
    Isaac Gravestock
    Janine Hoffart
    Renata S. Scalco
    Basil T. Darras
    Orphanet Journal of Rare Diseases, 17
12345