Treating neutropenia and neutrophil dysfunction in glycogen storage disease type Ib with an SGLT2 inhibitor von Wortmann, Saskia B., Van Hove, Johan L.K., Derks, Terry G.J., Chevalier, Nathalie, Knight, Vijaya, Koller, Andreas, Oussoren, Esmee, Mayr, Johannes A., van Spronsen, Francjan J., Lagler, Florian B., Gaughan, Sommer, Van Schaftingen, Emile, Veiga-da-Cunha, Maria

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Structural and functional rejuvenation of the aged brain by an approved anti-asthmatic drug von Marschallinger, Julia, Schäffner, Iris, Klein, Barbara, Gelfert, Renate, Rivera, Francisco J., Illes, Sebastian, Grassner, Lukas, Janssen, Maximilian, Rotheneichner, Peter, Schmuckermair, Claudia, Coras, Roland, Boccazzi, Marta, Chishty, Mansoor, Lagler, Florian B., Renic, Marija, Bauer, Hans-Christian, Singewald, Nicolas, Blümcke, Ingmar, Bogdahn, Ulrich, Couillard-Despres, Sebastien, Lie, D. Chichung, Abbracchio, Maria P., Aigner, Ludwig

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Development of a novel tool for individual treatment trials in mucopolysaccharidosis von Wiesinger, Anna-Maria, Bigger, Brian, Giugliani, Roberto, Lampe, Christina, Scarpa, Maurizio, Moser, Tobias, Kampmann, Christoph, Zimmermann, Georg, Lagler, Florian B

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Living with Pompe disease: results from a qualitative interview study with children and adolescents and their caregivers von Truninger, Moritz Ilan, Werner, Helene, Landolt, Markus Andreas, Hahn, Andreas, Hennermann, Julia B, Lagler, Florian B, Möslinger, Dorothea, Pfrimmer, Charlotte, Rohrbach, Marianne, Huemer, Martina

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

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Benefit‐Risk Assessment of Off‐Label Drug Use in Children: The Bravo Framework von Zanden, Tjitske M., Mooij, Miriam G., Vet, Nienke J., Neubert, Antje, Rascher, Wolfgang, Lagler, Florian B., Male, Christoph, Grytli, Helene, Halvorsen, Thomas, Hoog, Matthijs, Wildt, Saskia N.

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Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial von Wally, Verena, Hovnanian, Alain, Ly, Juliette, Buckova, Hana, Brunner, Victoria, Lettner, Thomas, Ablinger, Michael, Felder, Thomas K., Hofbauer, Peter, Wolkersdorfer, Martin, Lagler, Florian B., Hitzl, Wolfgang, Laimer, Martin, Kitzmüller, Sophie, Diem, Anja, Bauer, Johann W.

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Current knowledge, challenges and innovations in developmental pharmacology: A combined conect4children Expert Group and European Society for Developmental, Perinatal and Paediatri... von Smits, Anne, Annaert, Pieter, Cavallaro, Giacomo, De Cock, Pieter A. J. G., Wildt, Saskia N., Kindblom, Jenny M., Lagler, Florian B., Moreno, Carmen, Pokorna, Paula, Schreuder, Michiel F., Standing, Joseph F., Turner, Mark A., Vitiello, Benedetto, Zhao, Wei, Weingberg, Annelie‐Martina, Willmann, Raffaella, Anker, John, Allegaert, Karel

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The PompeQoL questionnaire: Development and validation of a new measure for children and adolescents with Pompe disease von Truninger, Moritz Ilan, Werner, Helene, Landolt, Markus Andreas, Hahn, Andreas, Hennermann, Julia B., Lagler, Florian B., Möslinger, Dorothea, Pfrimmer, Charlotte, Rohrbach, Marianne, Huemer, Martina

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Pharmacology of enalapril in children: a review von Smeets, Nori J.L., Schreuder, Michiel F., Dalinghaus, Michiel, Male, Christoph, Lagler, Florian B., Walsh, Jennifer, Laer, Stephanie, de Wildt, Saskia N.

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Innovative Treatments for Mucopolysaccharidoses von Lagler, Florian B.

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

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

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Long-term safety of sapropterin in paediatric and adult individuals with phenylalanine hydroxylase deficiency: Final results of the Kuvan® Adult Maternal Paediatric European Regist... von Feillet, François, Arnoux, Jean-Baptiste, Delgado, María Bueno, Burlina, Alberto, Chabrol, Brigitte, Kucuksayrac, Ece, Lagler, Florian B, Muntau, Ania C, Olsson, David, Paci, Sabrina, Rutsch, Frank, van Spronsen, Francjan J

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A survey of medicine use in children and adolescents in Austria von Rauch, Elisabeth, Lagler, Florian B., Herkner, Harald, Gall, Walter, Sauermann, Robert, Hetz, Silvia, Male, Christoph

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Efficacy and safety of sapropterin before and during pregnancy: Final analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER) maternal and Phenylketonuria Devel... von Feillet, François, Ficicioglu, Can, Lagler, Florian B., Longo, Nicola, Muntau, Ania C., Burlina, Alberto, Trefz, Friedrich K., Spronsen, Francjan J., Arnoux, Jean‐Baptiste, Lindstrom, Kristin, Lilienstein, Joshua, Clague, Gillian E., Rowell, Richard, Burton, Barbara K.

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Monitoring and integrated care coordination of patients with alpha-mannosidosis: A global Delphi consensus study von Guffon, Nathalie, Burton, Barbara K., Ficicioglu, Can, Magner, Martin, Gil-Campos, Mercedes, Lopez-Rodriguez, Monica A., Jayakar, Parul, Lund, Allan M., Tal, Galit, Garcia-Ortiz, Jose Elias, Stepien, Karolina M., Ellaway, Carolyn, Al-Hertani, Walla, Giugliani, Roberto, Cathey, Sara S., Hennermann, Julia B., Lampe, Christina, McNutt, Markey, Lagler, Florian B., Scarpa, Maurizio, Sutton, V. Reid, Muschol, Nicole

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

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

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

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