Individual Treatment Trials—Do Experts Know and Use This Option to Improve the Treatability of Mucopolysaccharidosis? by Anna-Maria Wiesinger, Hannah Strobl, Florian B. Lagler

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An artificial intelligence-based approach for identifying rare disease patients using retrospective electronic health records applied for Pompe disease by Simon Lin, Simon Lin, Jama Nateqi, Jama Nateqi, Rafael Weingartner-Ortner, Stefanie Gruarin, Hannes Marling, Vinzenz Pilgram, Florian B. Lagler, Florian B. Lagler, Elmar Aigner, Elmar Aigner, Alistair G. Martin

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The Inflammation in the Cytopathology of Patients With Mucopolysaccharidoses- Immunomodulatory Drugs as an Approach to Therapy by Anna-Maria Wiesinger, Anna-Maria Wiesinger, Brian Bigger, Brian Bigger, Roberto Giugliani, Maurizio Scarpa, Maurizio Scarpa, Tobias Moser, Christina Lampe, Christina Lampe, Christoph Kampmann, Florian B. Lagler, Florian B. Lagler

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RETRACTED: Wiesinger et al. An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis. <i>Pharmaceutics</i> 2023, <i>15</i>, 1565 by Anna-Maria Wiesinger, Brian Bigger, Roberto Giugliani, Christina Lampe, Maurizio Scarpa, Tobias Moser, Christoph Kampmann, Georg Zimmermann, Florian B. Lagler

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RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis by Anna-Maria Wiesinger, Brian Bigger, Roberto Giugliani, Christina Lampe, Maurizio Scarpa, Tobias Moser, Christoph Kampmann, Georg Zimmermann, Florian B. Lagler

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Extent, impact, and predictors of diagnostic delay in Pompe disease: A combined survey approach to unveil the diagnostic odyssey by Florian B. Lagler, Angelika Moder, Marianne Rohrbach, Julia Hennermann, Eugen Mengel, Seyfullah Gökce, Thomas Hundsberger, Kai M. Rösler, Nesrin Karabul, Martina Huemer

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Basal pharmacokinetic parameters of topically applied diacerein in pediatric patients with generalized severe epidermolysis bullosa simplex by Michael Ablinger, Thomas K. Felder, Monika Wimmer, Roland Zauner, Peter Hofbauer, Thomas Lettner, Martin Wolkersdorfer, Florian B. Lagler, Anja Diem, Johann W. Bauer, Verena Wally

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Enalapril and Enalaprilat Pharmacokinetics in Children with Heart Failure Due to Dilated Cardiomyopathy and Congestive Heart Failure after Administration of an Orodispersible Enala... by Stephanie Laeer, Willi Cawello, Bjoern B. Burckhardt, László Ablonczy, Milica Bajcetic, Johannes M. P. J. Breur, Michiel Dalinghaus, Christoph Male, Saskia N. de Wildt, Jörg Breitkreutz, Muhammed Faisal, Anne Keatley-Clarke, Ingrid Klingmann, Florian B. Lagler

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Orodispersible minitablets of enalapril for use in children with heart failure (LENA): Rationale and protocol for a multicentre pharmacokinetic bridging study and follow-up safety... by Milica Bajcetic, Saskia N. de Wildt, Michiel Dalinghaus, Jörg Breitkreutz, Ingrid Klingmann, Florian B. Lagler, Anne Keatley-Clarke, Johannes MPJ. Breur, Christoph Male, Ida Jovanovic, Andras Szatmári, László Ablonczy, Bjoern B. Burckhardt, Willi Cawello, Karl Kleine, Emina Obarcanin, Lucie Spatenkova, Vanessa Swoboda, Marijke van der Meulen, Peter Wagner, Jennifer Walsh, Stephanie Läer

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