Search Results - "Batsu, Isabela"

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency by Diaz, George A., Jones, Simon A., Scarpa, Maurizio, Mengel, Karl Eugen, Giugliani, Roberto, Guffon, Nathalie, Batsu, Isabela, Fraser, Patricia A., Li, Jing, Zhang, Qi, Ortemann-Renon, Catherine

“…To assess olipudase alfa enzyme replacement therapy for non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in children. This…”
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Treatment adherence and effect of concurrent statin intensity on the efficacy and safety of alirocumab in a real-life setting: results from ODYSSEY APPRISE by Banach, Maciej, López-Sendon, José Luis, Averna, Maurizio, Cariou, Bertrand, Loy, Megan, Manvelian, Garen, Batsu, Isabela, Poulouin, Yann, Gaudet, Daniel

“…The phase IIIb open-label ODYSSEY APPRISE study prospectively assessed the safety and efficacy of alirocumab (a proprotein convertase subtilisin/kexin type 9…”
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The diagnostic journey for patients with late-onset GM2 Gangliosidoses by Lopshire, Mariah C., Tifft, Cynthia, Burns, John, Gould, Rebecca, Zheng, Riliang, Batsu, Isabela

“…Late-onset forms of GM2 gangliosidosis-mainly, Tay-Sachs disease and Sandhoff disease-are under-recognized in clinical practice. In these rare lysosomal…”
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Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for Gaucher disease type 3: Phase 3 LEAP2MONO trial design by Heine, Walter, Batsu, Isabela, Minini, Pascal, Cherkasov, Dmitry

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AMETHIST: Baseline characteristics from a phase 3 trial of venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, in GM2-gangliosidoses and related diseases by Tifft, Cynthia J., Batsu, Isabela, Zheng, Riliang, Minini, Pascal

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Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for GM2 gangliosidosis and related diseases: Phase 3 AMETHIST trial design by Petrović, Miloš, Zheng, Riliang, Canabarro, Carla, Minini, Pascal, Batsu, Isabela

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ELISAFE: Baseline characteristics from an observational study to evaluate real-world safety of eliglustat in patients with Gaucher disease by Batsu, Isabela, Accomando, Beverly, Gaemers, Sebastiaan J.M., Sekulić, Davorka

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Long-term safety outcomes of eliglustat in patients with Gaucher disease: Prospective, multi-center, observational, post authorization safety sub-registry study by Giraldo, Pilar, Batsu, Isabela, Accomando, Beverly, Gaemers, Sebastiaan J.M., Cil, Sefika Uslu, Gouyette, Najat, Scarpa, Maurizio

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Gaucher disease type 3 patient journey: Results from interviews with neuronopathic Gaucher disease patients highlight the challenges of diagnosis and care by Refaei, Laith, Sekulic, Davorka, Doerr, Andrew, McCrone, Julia, Gould, Rebecca, Batsu, Isabela

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ELIKIDS: Baseline characteristics from the eliglustat substrate reduction therapy trial in children with Gaucher disease type 1 or type 3 by Giraldo, Pilar, Gokcay, Gulden, Batsu, Isabela, Kissell, Julie, Perichon, Maria G., Drelichman, Guillermo I.

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Olink® proteomic profiling of biofluids from patients with Gaucher disease type 3 to understand disease pathology by Levit, Mikhail, Bais, Preet, Krishnaswami, Pavithra, Batsu, Isabela, Zhang, Bailin, Klinger, Katherine W., Sardi, S. Pablo, Kayatekin, Can

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Changes in hematologic and visceral manifestations over time following imiglucerase initiation in Gaucher disease type 1 and type 3 pediatric patients in the ICGG Gaucher Registry by Mistry, Pramod K., Bianculli, Pablo, Batsu, Isabela, Heine, Walter, Oliveira-dos-Santos, Antonio, Minini, Pascal, Batista, Julie

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Safety and efficacy of alirocumab in a real-life setting: the ODYSSEY APPRISE study by Gaudet, Daniel, López-Sendón, José Luis, Averna, Maurizio, Bigot, Grégory, Banach, Maciej, Letierce, Alexia, Loy, Megan, Samuel, Rita, Manvelian, Garen, Batsu, Isabela, Henry, Patrick

“…To obtain safety and efficacy data of alirocumab, a proprotein convertase subtilisin/kexin type 9 inhibitor, in a real-life setting in high cardiovascular (CV)…”
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Two years of venglustat combined with imiglucerase shows continued positive effects on neurological features and brain connectivity in adults with Gaucher disease type 3 by Batsu, Isabela, Zheng, Riliang, Minini, Pascal, Cherkasov, Dmitry, Scott, David, Goker-Alpan, Ozlem, Hennermann, Julia B., Sakurai, Ken, Cox, Timothy M.

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Fine-motor functioning and its relevance to activities of daily living in adults with late-onset GM2 gangliosidosis: Analysis of baseline data of AMETHIST trial by Cox, Timothy M., Rodriguez, Maria B., Dyevre, Valérie, Msihid, Jerome, Gwaltney, Chad, Pulikottil-Jacob, Ruth, Zheng, Riliang, Minini, Pascal, Batsu, Isabela

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Qualitative Study of the Patient Experience with Venglustat for Gaucher Disease Type 3 in a Phase 2 Open-Label, Multicenter, Multinational Study (LEAP) by Schiffmann, Raphael, Mengel, Eugen, Wallace, Mary, Rochmann, Camille, Turnbull, James, Krupnick, Robert, Gwaltney, Chad, Pulikottil-Jacob, Ruth, Batsu, Isabela, Zheng, Riliang, Hamed, Alaa

“…Introduction Gaucher disease type 3 (GD3) is a genetic, progressive lysosomal storage disorder characterized by visceral manifestations and chronic neurologic…”
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One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency by Diaz, George A., Jones, Simon A., Scarpa, Maurizio, Mengel, Karl Eugen, Giugliani, Roberto, Guffon, Nathalie, Batsu, Isabela, Fraser, Patricia A., Li, Jing, Zhang, Qi, Ortemann-Renon, Catherine

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EFFICACY AND SAFETY OF ALIROCUMAB IN A REAL-LIFE SETTING IN PATIENTS WITH OR WITHOUT FAMILIAL HYPERCHOLESTEROLEMIA: THE ODYSSEY APPRISE STUDY by Gaudet, Daniel, López-Sendón, José Luis, Averna, Maurizio, Bigot, Gregory, Banach, Maciej, Letierce, Alexia, Loy, Megan, Samuel, Rita, Batsu, Isabela, Henry, Patrick

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One-year results of olipudase alfa enzyme replacement therapy in children with chronic visceral and neurovisceral acid sphingomyelinase deficiency by Diaz, George, Giugliani, Roberto, Guffon, Nathalie, Jones, Simon, Mengel, Eugen, Scarpa, Maurizio, Batsu, Isabela, Fraser, Patricia, Li, Jing, Zhang, Qi, Ortemann-Renon, Catherine

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The SYDNEY Device Study: A Multicenter, Randomized, Open-label Usability Study of a 2-mL Alirocumab Autoinjector Device by Frias, Juan Pablo, Koren, Michael J., Loizeau, Virginie, Merino-Trigo, Ana, Louie, Michael J., Raudenbush, Mary Alice, Batsu, Isabela

“…The proprotein convertase subtilisin/kexin type 9 inhibitor alirocumab has produced significant reductions in LDL-C at a dose of 300 mg q4w administered as 2…”
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