Newborn Screening and Emerging Therapies for X-Linked Adrenoleukodystrophy

被引：18

作者：

Moser, Ann B. ^{[1
]}

Fatemi, Ali ^{[1
]}

机构：

[1] Kennedy Krieger Inst, 707 N Broadway, Baltimore, MD 21205 USA

来源：

JAMA NEUROLOGY | 2018年 / 75卷 / 10期

关键词：

HEMATOPOIETIC-CELL TRANSPLANTATION;

D O I：

10.1001/jamaneurol.2018.1585

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

引用

页码：1175 / 1176

页数：2

共 50 条

[21] Adrenoleukodystrophy in the era of newborn screening
Eng, Liane
Regelmann, Molly O.
CURRENT OPINION IN ENDOCRINOLOGY DIABETES AND OBESITY, 2020, 27 (01) : 47 - 55
[22] Clinical course and endocrine dysfunction in X-linked adrenoleukodystrophy: A case series
Matos, Tania
Costa, Cristiana
Araujo, Alexandra Novais
do Vale, Sonia
ENDOCRINOLOGIA DIABETES Y NUTRICION, 2023, 70 (06): : 421 - 428
[23] Practical Approach to Longitudinal Neurologic Care of Adults With X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy
Kornbluh, Alexandra B.
Baldwin, Aaron
Fatemi, Ali
Vanderver, Adeline
Adang, Laura A.
Van Haren, Keith
Sampson, Jacinda
Eichler, Florian S.
Sadjadi, Reza
Engelen, Marc
Orthmann-Murphy, Jennifer L.
NEUROLOGY-GENETICS, 2024, 10 (05)
[24] Clinical aspects and adrenal functions in eleven Japanese children with X-linked adrenoleukodystrophy
Miyoshi, Yoko
Sakai, Norio
Hamada, Yusuke
Tachibana, Makiko
Hasegawa, Yasuhiro
Kiyohara, Yuki
Yamada, Hiroyuki
Murakami, Mari
Kondou, Hiroki
Kimura-Ohba, Shihoko
Mine, Junji
Sato, Tatsuharu
Kamio, Noriko
Ueda, Hitoshi
Suzuki, Yasuhiro
Shiomi, Masashi
Ohta, Hideaki
Shimozawa, Nobuyuki
Ozono, Keiichi
ENDOCRINE JOURNAL, 2010, 57 (11) : 965 - 972
[25] Clinical, biochemical, neuroimaging and molecular findings of X-linked Adrenoleukodystrophy patients in South China
Jiang, Min-yan
Cai, Yan-na
Liang, Cui-li
Peng, Min-zhi
Sheng, Hui-ying
Fan, Li-ping
Lin, Rui-zhu
Jiang, Hua
Huang, Yonglan
Liu, Li
METABOLIC BRAIN DISEASE, 2015, 30 (06) : 1439 - 1444
[26] Microglia damage precedes major myelin breakdown in X-linked adrenoleukodystrophy and metachromatic leukodystrophy
Bergner, Caroline G.
Van Der Meer, Franziska
Winkler, Anne
Wrzos, Claudia
Tuerkmen, Mevlude
Valizada, Emil
Fitzner, Dirk
Hametner, Simon
Hartmann, Christian
Pfeifenbring, Sabine
Stoltenburg-Didinger, Gisela
Brueck, Wolfgang
Nessler, Stefan
Stadelmann, Christine
GLIA, 2019, 67 (06) : 1196 - 1209
[27] A novel ABCD1 gene mutation in a Chinese patient with X-linked adrenoleukodystrophy
Cai, Yan-na
Jiang, Min-yan
Liang, Cui-li
Peng, Min-zhi
Cheng, Jing
Sheng, Hui-ying
Fan, Li-ping
Chen, Xi-qing
Liu, Li
JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM, 2015, 28 (5-6) : 725 - 729
[28] Evaluation of Neurofilament Light Chain as a Biomarker of Neurodegeneration in X-Linked Childhood Cerebral Adrenoleukodystrophy
Wang, Hongge
Davison, Matthew D.
Kramer, Martin L.
Qiu, Weiliang
Gladysheva, Tatiana
Chiang, Ruby M. S.
Kayatekin, Can
Nascene, David R.
Taghizadeh, Leyla A.
King, Carina J.
Nolan, Erin E.
Gupta, Ashish O.
Orchard, Paul J.
Lund, Troy C.
CELLS, 2022, 11 (05)
[29] Metabolic rerouting via SCD1 induction impacts X-linked adrenoleukodystrophy
Raas, Quentin
van de Beek, Malu-Clair
Forss-Petter, Sonja
Dijkstra, Inge M. E.
Deschiffart, Abigail
Freshner, Briana C.
Stevenson, Tamara J.
Jaspers, Yorrick R. J.
Nagtzaam, Liselotte
Wanders, Ronald J. A.
van Weeghel, Michel
Engelen-Lee, Joo-Yeon
Engelen, Marc
Eichler, Florian
Berger, Johannes
Bonkowsky, Joshua L.
Kemp, Stephan
JOURNAL OF CLINICAL INVESTIGATION, 2021, 131 (08)
[30] Early Signs of Visual Perception and Evoked Potentials in Radiologically Asymptomatic Boys With X-linked Adrenoleukodystrophy
Furushima, Wakana
Inagaki, Masumi
Gunji, Atsuko
Inoue, Yuki
Kaga, Makiko
Mizutani, Shuki
JOURNAL OF CHILD NEUROLOGY, 2009, 24 (08) : 927 - 935

← 1 2 3 4 5 →