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Loss of peroxisome function promotes oxidative stress-induced hematopoiesis 过氧化物酶体功能的丧失促进氧化应激诱导的造血作用 Schindhelm, Emma A.; Blake, Amada; Constans, Megan M.; Braaten, Kai; Thorn, Aly L.; Durose, Willa; Lorentson, Maggie; Gupta, Ashish O.; Orchard, Paul J.; Braverman, Nancy; Raymond, Gerald, V; Lund, Troy C. 分享 收藏
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A Phase 1 Study of Oral Vitamin D3 in Boys and Young Men With X-Linked Adrenoleukodystrophy X连锁肾上腺脑白质营养不良男孩和年轻男性口服维生素D3的1期研究 Van Haren, Keith P.; Cunanan, Kristen; Awani, Avni; Gu, Meng; Pena, Dalia; Chromik, Lindsay C.; Povazan, Michal; Rossi, Nicole C.; Goodman, Jordan; Sundaram, Vandana; Winterbottom, Jennifer; Raymond, Gerald, V; Cowan, Tina; Enns, Gregory M.; Waubant, Emmanuelle; Steinman, Lawrence; Barker, Peter B.; Spielman, Daniel; Fatemi, Ali 分享 收藏
International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy A Consensus-Based Approach Engelen, Marc; Van Ballegoij, Wouter J. C.; Mallack, Eric James; Van Haren, Keith P.; Kohler, Wolfgang; Salsano, Ettore; Van Trotsenburg, A. S. P.; Mochel, Fanny; Sevin, Caroline; Regelmann, Molly O.; Tritos, Nicholas A.; Halper, Alyssa; Lachmann, Robin H.; Davison, James; Raymond, Gerald V.; Lund, Troy C.; Orchard, Paul J.; Kuehl, Joern-Sven; Lindemans, Caroline A.; Caruso, Paul; Turk, Bela Rui; Moser, Ann B.; Vaz, Frederic M.; Ferdinandusse, Sacha; Kemp, Stephan; Fatemi, Ali; Eichler, Florian S.; Huffnagel, Irene C. 分享 收藏
Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy 治疗脑肾上腺脑白质营养不良: 同种异体移植和慢病毒基因治疗 Gupta, Ashish O.; Raymond, Gerald; Pierpont, Elizabeth, I; Kemp, Stephan; McIvor, R. Scott; Rayannavar, Arpana; Miller, Bradley; Lund, Troy C.; Orchard, Paul J. 分享 收藏
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Biomarker Identification, Safety, and Efficacy of High-Dose Antioxidants for Adrenomyeloneuropathy: a Phase II Pilot Study Casasnovas, Carlos; Ruiz, Montserrat; Schluter, Agatha; Naudi, Alba; Fourcade, Stephane; Veciana, Misericordia; Castaner, Sara; Alberti, Antonia; Bargallo, Nuria; Johnson, Maria; Raymond, Gerald, V; Fatemi, Ali; Moser, Ann B.; Villarroya, Francesc; Portero-Otin, Manuel; Artuch, Rafael; Pamplona, Reinald; Pujol, Aurora 分享 收藏
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Neurocognitive outcomes of intrathecal enzyme replacement therapy and transplant in Hurler syndrome Eisengart, Julie B.; Miller, Weston; Kaizer, Alex; Rudser, Kyle; King, Kelly; Ziegler, Richard S.; Pierpont, Elizabeth; Whitley, Chester; Raymond, Gerald; Tolar, Jakub; Lund, Troy; Shapiro, Elsa G.; Orchard, Paul 分享 收藏
Post-transplant adaptive function in childhood cerebral adrenoleukodystrophy Pierpont, Elizabeth I.; McCoy, Erin; King, Kelly E.; Ziegler, Rich S.; Shanley, Ryan; Nascene, David; Raymond, Gerald V.; Phelan, Rachel; Lund, Troy C.; Orchard, Paul J.; Miller, Weston P. 分享 收藏
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy Eichler, Florian; Duncan, Christine; Musolino, Patricia L.; Orchard, Paul J.; De Oliveira, Satiro; Thrasher, Adrian J.; Armant, Myriam; Dansereau, Colleen; Lund, Troy C.; Miller, Weston P.; Raymond, Gerald V.; Sankar, Raman; Shah, Ami J.; Sevin, Caroline; Gaspar, H. Bobby; Gissen, Paul; Amartino, Hernan; Bratkovic, Drago; Smith, Nicholas J. C.; Paker, Asif M.; Shamir, Esther; O'Meara, Tara; Davidson, David; Aubourg, Patrick; Williams, David A. 分享 收藏
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