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Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States Adang, Laura A.; Bonkowsky, Joshua L.; Boelens, Jaap Jan; Mallack, Eric; Ahrens-Nicklas, Rebecca; Bernat, John A.; Bley, Annette; Burton, Barbara; Darling, Alejandra; Eichler, Florian; Eklund, Erik; Emrick, Lisa; Escolar, Maria; Fatemi, Ali; Fraser, Jamie L.; Gaviglio, Amy; Keller, Stephanie; Patterson, Marc C.; Orchard, Paul; Orthmann-Murphy, Jennifer; Santoro, Jonathan D.; Schoels, Ludger; Sevin, Caroline; Srivastava, Isha N.; Rajan, Deepa; Rubin, Jennifer P.; Van Haren, Keith; Wasserstein, Melissa; Zerem, Ayelet; Fumagalli, Francesca; Laugwitz, Lucia; Vanderver, Adeline Share Save
Biomarker-based risk prediction for the onset of neuroinflammation in X-linked adrenoleukodystrophy Weinhofer, Isabelle; Rommer, Paulus; Gleiss, Andreas; Ponleitner, Markus; Zierfuss, Bettina; Waidhofer-Soellner, Petra; Fourcade, Stephane; Grabmeier-Pfisterhammer, Katharina; Reinert, Marie-Christine; Goepfert, Jens; Heine, Anne; Yska, Hemmo A. F.; Casasnovas, Carlos; Cantarin, Veronica; Bergner, Caroline G.; Mallack, Eric; Forss-Petter, Sonja; Aubourg, Patrick; Bley, Annette; Engelen, Marc; Eichler, Florian; Lund, Troy C.; Pujol, Aurora; Koehler, Wolfgang; Kuehl, Joern-Sven; Berger, Johannes Share Save
Early transgene expression of GALC enzyme in a phase I/II safety, tolerability and efficacy study of PBKR03 in infants with early infantile Krabbe disease (EIKD) (GALax-C) Al-Zaidy, Samiah A.; Wolf, Nicole I.; Jones, Simon; Mallack, Eric J.; Waldman, Amy; Rubin, Jennifer P.; Bonkowsky, Joshua L.; Aran, Adi; Nii, Yan G.; Nagilla, Pruthvi; Donahue, Dan; Giugliani, Roberto; Forman, Mark S.; Bernard, Genevieve Share Save
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. Share Save
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A phase 1/2 open-label, multicenter, dose ranging and confirmatory study to assess the safety, tolerability and efficacy of PBKR03 administered to pediatric subjects with early infantile Krabbe disease (globoid cell leukodystrophy; GALax-C) Mallack, Eric J.; Jones, Simon A.; Aran, Adi; Bernard, Genevieve; Bonkowsky, Joshua L.; Giugliani, Roberto; Rubin, Jennifer P.; Waldman, Amy T.; Wolf, Nicole I.; de Wall, Steve; McNeil, Elizabeth Share Save
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