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
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
收藏
页码:1175 / 1176
页数:2
相关论文
共 50 条
[41]   Childhood Cerebral X-Linked Adrenoleukodystrophy More Than 5 Years After Hematopoietic Cell Transplantation: The First Case From Serbia and Southeastern Europe [J].
Potic, Ana ;
Rovelli, Attilio M. ;
Uziel, Graziella ;
Kozic, Dusko ;
Mladenovic, Jelena ;
Milic-Rasic, Vedrana .
JOURNAL OF CHILD NEUROLOGY, 2010, 25 (12) :1542-1547
[42]   An International Survey of Allogeneic Hematopoietic Cell Transplantation for X-Linked Agammaglobulinemia [J].
Akira Nishimura ;
Ramya Uppuluri ;
Revathi Raj ;
Venkateswaran Vellaichamy Swaminathan ;
Yifei Cheng ;
Rolla F. Abu-Arja ;
Bin Fu ;
Alexandra Laberko ;
Michael H. Albert ;
Fabian Hauck ;
Giorgia Bucciol ;
Venetia Bigley ;
Suzanne Elcombe ;
Gaurav Kharya ;
Cornelis Jan H Pronk ;
Claudia Wehr ;
Bénédicte Neven ;
Klaus Warnatz ;
Isabelle Meyts ;
Tomohiro Morio ;
Andrew R. Gennery ;
Hirokazu Kanegane .
Journal of Clinical Immunology, 2023, 43 :1827-1839
[43]   An International Survey of Allogeneic Hematopoietic Cell Transplantation for X-Linked Agammaglobulinemia [J].
Nishimura, Akira ;
Uppuluri, Ramya ;
Raj, Revathi ;
Swaminathan, Venkateswaran Vellaichamy ;
Cheng, Yifei ;
Abu-Arja, Rolla F. ;
Fu, Bin ;
Laberko, Alexandra ;
Albert, Michael H. ;
Hauck, Fabian ;
Bucciol, Giorgia ;
Bigley, Venetia ;
Elcombe, Suzanne ;
Kharya, Gaurav ;
Pronk, Cornelis Jan H. ;
Wehr, Claudia ;
Neven, Benedicte ;
Warnatz, Klaus ;
Meyts, Isabelle ;
Morio, Tomohiro ;
Gennery, Andrew R. ;
Kanegane, Hirokazu .
JOURNAL OF CLINICAL IMMUNOLOGY, 2023, 43 (08) :1827-1839
[44]   Hematopoietic cell transplantation for asymptomatic X-linked lymphoproliferative syndrome type 1 [J].
Tamura, Akihiro ;
Uemura, Suguru ;
Yamamoto, Nobuyuki ;
Saito, Atsuro ;
Kozaki, Aiko ;
Kishimoto, Kenji ;
Ishida, Toshiaki ;
Hasegawa, Daiichiro ;
Hiroki, Haruka ;
Okano, Tsubasa ;
Imai, Kohsuke ;
Morio, Tomohiro ;
Kanegane, Hirokazu ;
Kosaka, Yoshiyuki .
ALLERGY ASTHMA AND CLINICAL IMMUNOLOGY, 2018, 14
[45]   Hematopoietic cell transplantation for asymptomatic X-linked lymphoproliferative syndrome type 1 [J].
Akihiro Tamura ;
Suguru Uemura ;
Nobuyuki Yamamoto ;
Atsuro Saito ;
Aiko Kozaki ;
Kenji Kishimoto ;
Toshiaki Ishida ;
Daiichiro Hasegawa ;
Haruka Hiroki ;
Tsubasa Okano ;
Kohsuke Imai ;
Tomohiro Morio ;
Hirokazu Kanegane ;
Yoshiyuki Kosaka .
Allergy, Asthma & Clinical Immunology, 14
[46]   Deficiency in X-linked inhibitor of apoptosis protein promotes susceptibility to microbial triggers of intestinal inflammation [J].
Strigli, Anne ;
Gopalakrishnan, Shreya ;
Zeissig, Yvonne ;
Basic, Marijana ;
Wang, Jun ;
Schwerd, Tobias ;
Doms, Shauni ;
Peuker, Kenneth ;
Hartwig, Jelka ;
Harder, Juergen ;
Hoenscheid, Pia ;
Arnold, Philipp ;
Kurth, Thomas ;
Rost, Fabian ;
Petersen, Britt-Sabina ;
Forster, Michael ;
Franke, Andre ;
Kelsen, Judith R. ;
Rohlfs, Meino ;
Klein, Christoph ;
Muise, Aleixo M. ;
Warner, Neil ;
Nambu, Ryusuke ;
Mayerle, Julia ;
Toeroek, Helga-Paula ;
Linkermann, Andreas ;
Muders, Michael H. ;
Baretton, Gustavo B. ;
Hampe, Jochen ;
Aust, Daniela E. ;
Baines, John F. ;
Bleich, Andre ;
Zeissig, Sebastian .
SCIENCE IMMUNOLOGY, 2021, 6 (65)
[47]   A Novel BTK Gene Mutation in a Child With Atypical X-Linked Agammaglobulinemia and Recurrent Hemophagocytosis: A Case Report [J].
Han, Shu-Ping ;
Line, Yung-Feng ;
Weng, Hui-Ping ;
Tsai, Shih-Feng ;
Fu, Lin-Shien .
FRONTIERS IN IMMUNOLOGY, 2019, 10
[48]   Successful treatment of post-transplant thrombocytopenia with romiplostim in a pediatric patient with X-linked chronic granulomatous disease [J].
Buchbinder, David ;
Hsieh, Loan ;
Krance, Robert ;
Nugent, Diane J. .
PEDIATRIC TRANSPLANTATION, 2014, 18 (07) :E252-E254
[49]   Clinical, Laboratory Features and Clinical Courses of Patients with Wiskott Aldrich Syndrome and X-linked Thrombocytopenia-A single center study [J].
Bildik, Hacer Neslihan ;
Cagdas, Deniz ;
Kura, Aysenur Ozturk ;
Halacli, Sevil Oskay ;
Sanal, Ozden ;
Tezcan, Ilhan .
IMMUNOLOGICAL INVESTIGATIONS, 2022, 51 (05) :1272-1283
[50]   Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation [J].
de la Morena, M. Teresa ;
Leonard, David ;
Torgerson, Troy R. ;
Cabral-Marques, Otavio ;
Slatter, Mary ;
Aghamohammadi, Asghar ;
Chandra, Sharat ;
Murguia-Favela, Luis ;
Bonilla, Francisco A. ;
Kanariou, Maria ;
Damrongwatanasuk, Rongras ;
Kuo, Caroline Y. ;
Dvorak, Christopher C. ;
Meyts, Isabelle ;
Chen, Karin ;
Kobrynski, Lisa ;
Kapoor, Neena ;
Richter, Darko ;
DiGiovanni, Daniela ;
Dhalla, Fatima ;
Farmaki, Evangelia ;
Speckmann, Carsten ;
Espanol, Teresa ;
Shcherbina, Anna ;
Hanson, Imelda Celine ;
Litzman, Jiri ;
Routes, John M. ;
Wong, Melanie ;
Fuleihan, Ramsay ;
Seneviratne, Suranjith L. ;
Small, Trudy N. ;
Janda, Ales ;
Bezrodnik, Liliana ;
Seger, Reinhard ;
Raccio, Andrea Gomez ;
Edgar, J. David M. ;
Chou, Janet ;
Abbott, Jordan K. ;
van Montfrans, Joris ;
Gonzalez-Granado, Luis Ignacio ;
Bunin, Nancy ;
Kutukculer, Necil ;
Gray, Paul ;
Seminario, Gisela ;
Pasic, Srdjan ;
Aquino, Victor ;
Wysocki, Christian ;
Abolhassani, Hassan ;
Dorsey, Morna ;
Cunningham-Rundles, Charlotte .
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2017, 139 (04) :1282-1292