Selexipag: an oral, selective prostacyclin receptor agonist for the treatment of pulmonary arterial hypertension von SIMONNEAU, Gérald, TORBICKI, Adam, GIORGINO, Ruben, LANG, Irene M, HOEPER, Marius M, DELCROIX, Marion, KARLOCAI, Kristóf, GALIE, Nazzareno, DEGANO, Bruno, BONDERMAN, Diana, KURZYNA, Marcin, EFFICACE, Michela

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High prevalence of asymptomatic vertebral fractures in post-menopausal women receiving chronic glucocorticoid therapy: A cross-sectional outpatient study von Angeli, Alberto, Guglielmi, Giuseppe, Dovio, Andrea, Capelli, Giovanni, de Feo, Daniela, Giannini, Sandro, Giorgino, Ruben, Moro, Luigi, Giustina, Andrea

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High prevalence of hypovitaminosis D in female type 2 diabetic population von Isaia, G, Giorgino, R, Adami, S

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Miglustat therapy in type 1 Gaucher disease: Clinical and safety outcomes in a multicenter retrospective cohort study von Kuter, David J., Mehta, Atul, Hollak, Carla E.M., Giraldo, Pilar, Hughes, Derralynn, Belmatoug, Nadia, Brand, Monika, Muller, Audrey, Schaaf, Berthold, Giorgino, Ruben, Zimran, Ari

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Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study von Cox, Timothy M, Amato, Dominick, Hollak, Carla Em, Luzy, Cecile, Silkey, Mariabeth, Giorgino, Ruben, Steiner, Robert D

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The effect of age, weight, and lifestyle factors on calcaneal quantitative ultrasound: the ESOPO study von ADAMI, Silvano, GIANNINI, Sandro, GIORGINO, Ruben, ISAIA, Giancarlo, MAGGI, Stefania, SINIGAGLIA, Luigi, FILIPPONI, Paolo, CREPALDI, Gaetano, DI MUNNO, Ombretta

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First-in-human single-dose study of nizubaglustat, a dual inhibitor of ceramide glucosyltransferase and non-lysosomal glucosylceramidase: Safety, tolerability, pharmacokinetics, an... von Paquet Luzy, Cecile, Doppler, Emilie, Polasek, Thomas M., Giorgino, Ruben

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Miglustat in adult and juvenile patients with Niemann–Pick disease type C: Long-term data from a clinical trial von Wraith, James E., Vecchio, Darleen, Jacklin, Elizabeth, Abel, Larry, Chadha-Boreham, Harbajan, Luzy, Cécile, Giorgino, Ruben, Patterson, Marc C.

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GM1‐gangliosidosis: The caregivers' assessments of symptom impact and most important symptoms to treat von Bingaman, Amanda, Waggoner, Christine, Andrews, Sara M., Pangonis, Diana, Trad, Marie, Giugliani, Roberto, Giorgino, Ruben, Jarnes, Jeanine, Vakili, Rojan, Ballard, Victoria, Peay, Holly L.

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Elsiglutide in the primary prevention of chemotherapy (CT)-induced diarrhea in patients with colorectal cancer (CRC) receiving 5-fluorouracil (5-FU)-based CT: A multinational, rand... von Karthaus, Meinolf, Fedyanin, Mikhail, Bondarenko, Igor, Chessari, Salvatore, Spezia, Riccardo, Giorgino, Ruben

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Body weight response with anamorelin in advanced non-small cell lung cancer (NSCLC) patients with anorexia/cachexia: Pooled analysis of two phase III trials von Currow, David Christopher, Temel, Jennifer S., Abernethy, Amy Pickar, Friend, John, Giorgino, Ruben

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Long-Term Miglustat Therapy in Children With Niemann-Pick Disease Type C von Patterson, Marc C., Vecchio, Darleen, Jacklin, Elizabeth, Abel, Larry, Chadha-Boreham, Harbajan, Luzy, Cecile, Giorgino, Ruben, Wraith, James E.

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Efficacy of anamorelin in cachectic patients with non-small cell lung cancer (NSCLC) and low BMI (< 20 kg/m 2 ): Post-hoc analysis of two phase III studies von Currow, David Christopher, Temel, Jennifer S., Abernethy, Amy Pickar, Giorgino, Ruben, Friend, John, Fearon, Kenneth

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Anamorelin in cachectic patients with non-small cell lung cancer (NSCLC) and low BMI (< 20 kg/m 2 ): A pooled efficacy data analysis of two phase 3 studies von Currow, David Christopher, Temel, Jennifer S., Giorgino, Ruben, Abernethy, Amy Pickar, Friend, John, Fearon, Kenneth

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Pracinostat plus azacitidine in older patients with newly diagnosed acute myeloid leukemia: results of a phase 2 study von Garcia-Manero, Guillermo, Abaza, Yasmin, Takahashi, Koichi, Medeiros, Bruno C., Arellano, Martha, Khaled, Samer K., Patnaik, Mrinal, Odenike, Olatoyosi, Sayar, Hamid, Tummala, Mohan, Patel, Prapti, Maness-Harris, Lori, Stuart, Robert, Traer, Elie, Karamlou, Kasra, Yacoub, Abdulraheem, Ghalie, Richard, Giorgino, Ruben, Atallah, Ehab

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25(OH)D Serum Levels Decline With Age Earlier in Women Than in Men and Less Efficiently Prevent Compensatory Hyperparathyroidism in Older Adults von Maggio, Dario, Cherubini, Antonio, Lauretani, Fulvio, Russo, Roberto Cosimo, Bartali, Benedetta, Pierandrei, Marco, Ruggiero, Carmelinda, Macchiarulo, Maria Carmela, Giorgino, Ruben, Minisola, Salvatore, Ferrucci, Luigi

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Miglustat Therapy in Type 1 Gaucher Disease: Long-Term Treatment Experience From a Multicenter, Retrospective Cohort Study von Kuter, David J., Mehta, Atul, Hollak, Carla, Giraldo, Pilar, Hughes, Derralynn, Belmatoug, Nadia, Brand, Monika, Muller, Audrey, Schaaf, Berthold, Giorgino, Ruben, Zimran, Ari

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Baseline data from a prospective international disease registry for Niemann-Pick disease type C von Patterson, Marc, Mengel, Eugen, Wraith, Ed, Wijburg, Frits, Vanier, Marie, Schwierin, Barbara, Muller, Audrey, Silkey, Mariabeth, Giorgino, Ruben, Pineda, Mercedes

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154. Disease stability in patients with Niemann–Pick disease type C treated with miglustat von Wraith, Ed, Vecchio, Darlenn, Jacklin, Elizabeth, Luzy, Cicile, Giorgino, Ruben, Patterson, Marc

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METHODS OF DETERMINING AND PROVIDING CLINICALLY MEANINGFUL IMPROVEMENTS ON PATIENT REPORTED OUTCOMES IN ANOREXIA / CACHEXIA PATIENTS von CHESSARI, Salvatore, GIORGINO, Ruben, PALMESINO, Elena

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