Systemic corticosteroids for the treatment of acute episodes of rhabdomyolysis in lipin‐1‐deficient patients by Tuchmann‐Durand, Caroline, Roda, Célina, Renard, Perrine, Mortamet, Guillaume, Bérat, Claire‐Marine, Altenburger, Lucile, Larauz, Marie Hug, Thevenet, Eloise, Cottart, Charles‐Henry, Moulin, Florence, Bouchereau, Juliette, Brassier, Anais, Arnoux, Jean‐Baptiste, Schiff, Manuel, Bednarek, Nathalie, Lamireau, Delphine, Garros, Alexa, Mention, Karine, Cano, Aline, Finger, Lionel, Pelosi, Michele, Brochet, Cécile Sergent, Caccavelli, Laure, Raphalen, Jean‐Herlé, Renolleau, Sylvain, Oualha, Mehdi, Lonlay, Pascale

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Circulatory response to exercise relative to oxygen uptake assessed in the follow-up of patients with fatty acid beta-oxidation disorders by Imbard, Apolline, de Calbiac, Hortense, Le Guillou, Edouard, Laforêt, Pascal, Schiff, Manuel, Brassier, Anaïs, Thevenet, Elise, Pontoizeau, Clément, Lefrère, Bertrand, Ottolenghi, Chris, Lebigot, Elise, Gaignard, Pauline, Gobin, Stéphanie, Acquaviva-Bourdain, Cécile, Benoist, Jean-François, Tuchmann-Durand, Caroline, Legendre, Antoine, de Lonlay, Pascale

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Administration of gamma‐hydroxybutyrate instead of beta‐hydroxybutyrate to a liver transplant recipient suffering from propionic acidemia and cardiomyopathy: A case report on a med... by Tuchmann‐Durand, Caroline, Thevenet, Eloise, Moulin, Florence, Lesage, Fabrice, Bouchereau, Juliette, Oualha, Mehdi, Khraiche, Diala, Brassier, Anaïs, Wicker, Camille, Gobin‐Limballe, Stéphanie, Arnoux, Jean‐Baptiste, Lacaille, Florence, Wicart, Clotilde, Coat, Bruno, Schlattler, Joel, Cisternino, Salvatore, Renolleau, Sylvain, Secretan, Philippe‐Henri, De Lonlay, Pascale

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Preclinical Assessment for Gene Addition Strategy in Familial Hemophagocytic Lymphohistiocytosis Related to Munc 13-4 Deficiency by Lagresle-Peyrou, Chantal, Diana, Jean-Sebastien, Magrin, Elisa, Aussel, Clotilde, Sorel, Natael, Aslan, Yetki, Roudaut, Cécile, Schwartz, Eden, Gabrion, Aurélie, Tuchmann-Durand, Caroline, Lefrere, Francois, Galy, Anne, Moshous, Despina, Cavazzana, Marina

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Gene Therapy for Artemis-SCID and Artemis-Leaky-SCID Patients: Preliminary Results of the French Artegene Phase I/II Clinical Trial by Diana, Jean-Sebastien, Moshous, Despina, Castelle, Martin, Bessot, Boris, Magrin, Elisa, Chbihi, Marwa, Bastard, Paul, Girardot, Alice, Roudaut, Cecile, Mollet, Chloe, Aussel, Clotilde, Tuchmann-Durand, Caroline, Schwartz, Eden, Neth, Olaf, Olbrich, Peter, d'Alba, Irene, Riviere, Jacques, Oualha, Medhi, Joseph, Laure, Neven, Benedicte, De Villartay, Jean-Pierre, Galy, Anne, Lagresle-Peyrou, Chantal, Cavazzana, Marina

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Ex Vivo Generation of Human T-Cell Progenitors from Cord Blood CD34+ Cells Can Accelerate Immune Reconstitution after Umbilical Cord Blood Transplantation in Adult Patient with Hem... by Forcade, Edouard, Huynh, Anne, Magrin, Elisa, Diana, Jean-Sebastien, Schwartz, Eden, Aussel, Clotilde, Gabrion, Aurélie, Maulet, Axelle, Girardot, Alice, Ternaux, Brigitte, Tuchmann-Durand, Caroline, Baek, Jimni, Lhermitte, Ludovic, Touzart, Aurore, Asnafi, Vahid, Bouazza, Naim, Bauquet, Aurelie, Soheili, Shabi, Negre, Olivier, André, Isabelle, Treluyer, Jean-Marc, Hermine, Olivier, Cavazzana, Marina

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Efficacy of oral manganese and D-galactose therapy in a patient bearing a novel TMEM165 variant by Durin, Zoé, Raynor, Alexandre, Fenaille, François, Cholet, Sophie, Vuillaumier-Barrot, Sandrine, Alili, Jean-Meidi, Poupon, Joël, Oussedik, Nouzha Djebrani, Tuchmann-Durand, Caroline, Attali, Jennifer, Touzé, Romain, Dupré, Thierry, Lebredonchel, Elodie, Akaffou, Marlyse Angah, Legrand, Dominique, de Lonlay, Pascale, Bruneel, Arnaud, Foulquier, François

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Hydroxychloroquine sulfate: A novel treatment for lipin-1 deficiency? by Renard, Perrine, Caccavelli, Laure, Legendre, Antoine, Tuchmann-Durand, Caroline, Balakirouchenane, David, Blanchet, Benoit, Narjoz, Céline, Straube, Marjolène, Hubas, Arnaud, Garros, Alexa, Mention, Karine, Bednarek, Nathalie, Goudin, Nicolas, Broissand, Christine, Schlatter, Joel, Cisternino, Salvatore, Cagnard, Nicolas, van Endert, Peter, Diana, Julien, de Calbiac, Hortense, de Lonlay, Pascale

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Formulation and stability study of hydroxychloroquine sulfate oral suspensions by El Mershati, Sarah, Thouvenin, Agathe, Secretan, Philippe-Henri, De Lonlay, Pascale, Tuchmann-Durand, Caroline, Cisternino, Salvatore, Schlatter, Joël

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Compromised mitochondrial quality control triggers lipin1-related rhabdomyolysis by Hamel, Yamina, Mauvais, François-Xavier, Madrange, Marine, Renard, Perrine, Lebreton, Corinne, Nemazanyy, Ivan, Pellé, Olivier, Goudin, Nicolas, Tang, Xiaoyun, Rodero, Mathieu P., Tuchmann-Durand, Caroline, Nusbaum, Patrick, Brindley, David N., van Endert, Peter, de Lonlay, Pascale

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Administration of gamma-hydroxybutyrate instead of beta-hydroxybutyrate to a liver transplant recipient suffering from propionic acidemia and cardiomyopathy: A case report on a med... by Tuchmann-Durand, Caroline, Thevenet, Eloise, Moulin, Florence, Lesage, Fabrice, Bouchereau, Juliette, Oualha, Mehdi, Khraiche, Diala, Brassier, Anaïs, Wicker, Camille, Gobin-Limballe, Stéphanie, Arnoux, Jean-Baptiste, Lacaille, Florence, Wicart, Clotilde, Coat, Bruno, Schlattler, Joel, Cisternino, Salvatore, Renolleau, Sylvain, Secretan, Philippe-Henri, De Lonlay, Pascale

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