يعرض 1 - 20 نتائج من 40 نتيجة بحث عن '"Batsu, Isabela"', وقت الاستعلام: 0.50s تنقيح النتائج
  1. 1
    Academic Journal
    The diagnostic journey for patients with late-onset GM2 Gangliosidoses

    المؤلفون: Lopshire, Mariah C., Tifft, Cynthia, Burns, John, Gould, Rebecca, Zheng, Riliang, Batsu, Isabela

    المساهمون: Sanofi Genzyme, Sanofi

    المصدر: Molecular Genetics and Metabolism Reports ; volume 37, page 101014 ; ISSN 2214-4269

    الاتاحة: http://dx.doi.org/10.1016/j.ymgmr.2023.101014
    https://api.elsevier.com/content/article/PII:S2214426923000605?httpAccept=text/xml
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  2. 2
    Academic Journal
    One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

    المؤلفون: 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

    المصدر: Genetics in Medicine ; volume 24, issue 10, page 2209 ; ISSN 1098-3600

    الاتاحة: https://doi.org/10.1016/j.gim.2022.08.011
    https://api.elsevier.com/content/article/PII:S1098360022009029?httpAccept=text/xml
    https://api.elsevier.com/content/article/PII:S1098360022009029?httpAccept=text/plain

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  3. 3
    Academic Journal
    Qualitative Study of the Patient Experience with Venglustat for Gaucher Disease Type 3 in a Phase 2 Open-Label, Multicenter, Multinational Study (LEAP).

    المؤلفون: Schiffmann, Raphael, Mengel, Eugen, Wallace, Mary, Rochmann, Camille, Turnbull, James, Krupnick, Robert, Gwaltney, Chad, Pulikottil-Jacob, Ruth, Batsu, Isabela, Zheng, Riliang, Hamed, Alaa

    المصدر: Advances in Therapy; Jul2024, Vol. 41 Issue 7, p2907-2923, 17p


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  4. 4
    Academic Journal
    Olink® proteomic profiling of biofluids from patients with Gaucher disease type 3 to understand disease pathology

    المؤلفون: Levit, Mikhail, Bais, Preet, Krishnaswami, Pavithra, Batsu, Isabela, Zhang, Bailin, Klinger, Katherine W., Sardi, S. Pablo, Kayatekin, Can

    المصدر: Molecular Genetics and Metabolism ; volume 141, issue 2, page 107937 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2023.107937
    https://api.elsevier.com/content/article/PII:S109671922300567X?httpAccept=text/xml
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  5. 5
    Academic Journal
    Long-term safety outcomes of eliglustat in patients with Gaucher disease: Prospective, multi-center, observational, post authorization safety sub-registry study

    المؤلفون: Giraldo, Pilar, Batsu, Isabela, Accomando, Beverly, Gaemers, Sebastiaan J.M., Cil, Sefika Uslu, Gouyette, Najat, Scarpa, Maurizio

    المصدر: Molecular Genetics and Metabolism ; volume 141, issue 2, page 107844 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2023.107844
    https://api.elsevier.com/content/article/PII:S1096719223004742?httpAccept=text/xml
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  6. 6
    Academic Journal
    One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

    المؤلفون: Diaz, George A., Jones, Simon A., Scarpa, Maurizio, Mengel, Karl Eugen, Giugliani, Roberto, Guffon, Nathalie, Batsu, Isabela, Fraser, Patricia A., Jing, Li, Qi, Zhang, Renon, Catherine Ortemann

    مصطلحات موضوعية: Terapia de reposição de enzimas, Doenças de Niemann-Pick, Criança

    وصف الملف: application/pdf

    Relation: Genetics in medicine. New York. Vol. 23 (2021), p. 1543–1550.; http://hdl.handle.net/10183/234517; 001135853

    الاتاحة: http://hdl.handle.net/10183/234517

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  7. 7
    Academic Journal
    Safety and efficacy of alirocumab in a real-life setting: the ODYSSEY APPRISE study

    المؤلفون: Gaudet, Daniel, López-Sendón, José Luis, Averna, Maurizio, Bigot, Grégory, Banach, Maciej, Letierce, Alexia, Loy, Megan, Samuel, Rita, Manvelian, Garen, Batsu, Isabela, Henry, Patrick

    المساهمون: Sanofi and Regeneron Pharmaceuticals, Inc

    المصدر: European Journal of Preventive Cardiology ; volume 28, issue 17, page 1864-1872 ; ISSN 2047-4873 2047-4881

    الاتاحة: https://doi.org/10.1093/eurjpc/zwaa097
    https://academic.oup.com/eurjpc/article-pdf/28/17/1864/42379955/zwaa097.pdf

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  8. 8
    Academic Journal
    The SYDNEY Device Study: A Multicenter, Randomized, Open-label Usability Study of a 2-mL Alirocumab Autoinjector Device

    المؤلفون: Frias, Juan Pablo, Koren, Michael J., Loizeau, Virginie, Merino-Trigo, Ana, Louie, Michael J., Raudenbush, Mary Alice, Batsu, Isabela

    المساهمون: Regeneron Pharmaceuticals, Sanofi

    المصدر: Clinical Therapeutics ; volume 42, issue 1, page 94-107.e5 ; ISSN 0149-2918

    الاتاحة: http://dx.doi.org/10.1016/j.clinthera.2019.11.008
    https://api.elsevier.com/content/article/PII:S0149291819305697?httpAccept=text/xml
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  9. 9
    Academic Journal
    EFFICACY AND SAFETY OF ALIROCUMAB IN A REAL-LIFE SETTING IN PATIENTS WITH OR WITHOUT FAMILIAL HYPERCHOLESTEROLEMIA: THE ODYSSEY APPRISE STUDY

    المؤلفون: Gaudet, Daniel, López-Sendón, José Luis, Averna, Maurizio, Bigot, Gregory, Banach, Maciej, Letierce, Alexia, Loy, Megan, Samuel, Rita, Batsu, Isabela, Henry, Patrick

    المصدر: Journal of the American College of Cardiology ; volume 75, issue 11, page 1958 ; ISSN 0735-1097

    الاتاحة: http://dx.doi.org/10.1016/s0735-1097(20)32585-7
    https://api.elsevier.com/content/article/PII:S0735109720325857?httpAccept=text/xml
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  10. 10
    Academic Journal
    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

    المؤلفون: Cox, Timothy M., Rodriguez, Maria B., Dyevre, Valérie, Msihid, Jerome, Gwaltney, Chad, Pulikottil-Jacob, Ruth, Zheng, Riliang, Minini, Pascal, Batsu, Isabela

    المصدر: Molecular Genetics and Metabolism ; volume 138, issue 2, page 107063 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2022.107063
    https://api.elsevier.com/content/article/PII:S1096719222005418?httpAccept=text/xml
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  11. 11
    Academic Journal
    ELIKIDS: Baseline characteristics from the eliglustat substrate reduction therapy trial in children with Gaucher disease type 1 or type 3

    المؤلفون: Giraldo, Pilar, Gokcay, Gulden, Batsu, Isabela, Kissell, Julie, Perichon, Maria G., Drelichman, Guillermo I.

    المصدر: Molecular Genetics and Metabolism ; volume 138, issue 2, page 107113 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2022.107113
    https://api.elsevier.com/content/article/PII:S1096719222005911?httpAccept=text/xml
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  12. 12
    Academic Journal
    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

    المؤلفون: Mistry, Pramod K., Bianculli, Pablo, Batsu, Isabela, Heine, Walter, Oliveira-dos-Santos, Antonio, Minini, Pascal, Batista, Julie

    المصدر: Molecular Genetics and Metabolism ; volume 138, issue 2, page 107225 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2022.107225
    https://api.elsevier.com/content/article/PII:S109671922200703X?httpAccept=text/xml
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  13. 13
    Academic Journal
    Gaucher disease type 3 patient journey: Results from interviews with neuronopathic Gaucher disease patients highlight the challenges of diagnosis and care

    المؤلفون: Refaei, Laith, Sekulic, Davorka, Doerr, Andrew, McCrone, Julia, Gould, Rebecca, Batsu, Isabela

    المصدر: Molecular Genetics and Metabolism ; volume 138, issue 2, page 107286 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2022.107286
    https://api.elsevier.com/content/article/PII:S1096719222007648?httpAccept=text/xml
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  14. 14
    Academic Journal
    AMETHIST: Baseline characteristics from a phase 3 trial of venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, in GM2-gangliosidoses and related diseases

    المؤلفون: Tifft, Cynthia J., Batsu, Isabela, Zheng, Riliang, Minini, Pascal

    المصدر: Molecular Genetics and Metabolism ; volume 138, issue 2, page 107335 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2022.107335
    https://api.elsevier.com/content/article/PII:S1096719222008137?httpAccept=text/xml
    https://api.elsevier.com/content/article/PII:S1096719222008137?httpAccept=text/plain

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  15. 15
    Academic Journal
    Two years of venglustat combined with imiglucerase shows continued positive effects on neurological features and brain connectivity in adults with Gaucher disease type 3

    المؤلفون: Batsu, Isabela, Zheng, Riliang, Minini, Pascal, Cherkasov, Dmitry, Scott, David, Goker-Alpan, Ozlem, Hennermann, Julia B., Sakurai, Ken, Cox, Timothy M.

    المصدر: Molecular Genetics and Metabolism ; volume 138, issue 2, page 107024 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2022.107024
    https://api.elsevier.com/content/article/PII:S1096719222005029?httpAccept=text/xml
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  16. 16
    Academic Journal
    Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for Gaucher disease type 3: Phase 3 LEAP2MONO trial design

    المؤلفون: Heine, Walter, Batsu, Isabela, Minini, Pascal, Cherkasov, Dmitry

    المصدر: Molecular Genetics and Metabolism ; volume 138, issue 2, page 107144 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2022.107144
    https://api.elsevier.com/content/article/PII:S1096719222006229?httpAccept=text/xml
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  17. 17
    Academic Journal
    ELISAFE: Baseline characteristics from an observational study to evaluate real-world safety of eliglustat in patients with Gaucher disease

    المؤلفون: Batsu, Isabela, Accomando, Beverly, Gaemers, Sebastiaan J.M., Sekulić, Davorka

    المصدر: Molecular Genetics and Metabolism ; volume 135, issue 2, page S21-S22 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2021.11.036
    https://api.elsevier.com/content/article/PII:S1096719221008544?httpAccept=text/xml
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  18. 18
    Academic Journal
    Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for GM2 gangliosidosis and related diseases: Phase 3 AMETHIST trial design

    المؤلفون: Petrović, Miloš, Zheng, Riliang, Canabarro, Carla, Minini, Pascal, Batsu, Isabela

    المصدر: Molecular Genetics and Metabolism ; volume 135, issue 2, page S96-S97 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/j.ymgme.2021.11.252
    https://api.elsevier.com/content/article/PII:S1096719221010702?httpAccept=text/xml
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  19. 19
    Academic Journal
    One-year results of olipudase alfa enzyme replacement therapy in children with chronic visceral and neurovisceral acid sphingomyelinase deficiency

    المؤلفون: Diaz, George, Giugliani, Roberto, Guffon, Nathalie, Jones, Simon, Mengel, Eugen, Scarpa, Maurizio, Batsu, Isabela, Fraser, Patricia, Li, Jing, Zhang, Qi, Ortemann-Renon, Catherine

    المصدر: Molecular Genetics and Metabolism ; volume 132, page S115 ; ISSN 1096-7192

    الاتاحة: http://dx.doi.org/10.1016/s1096-7192(21)00260-2
    https://api.elsevier.com/content/article/PII:S1096719221002602?httpAccept=text/xml
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  20. 20
    Academic Journal
    Treatment adherence and effect of concurrent statin intensity on the efficacy and safety of alirocumab in a real-life setting: results from ODYSSEY APPRISE.

    المؤلفون: Banach, Maciej, López-Sendon, José Luis, Averna, Maurizio, Cariou, Bertrand, Loy, Megan, Manvelian, Garen, Batsu, Isabela, Poulouin, Yann, Gaudet, Daniel

    المصدر: Archives of Medical Science; 2022, Vol. 18 Issue 2, p285-292, 8p

    مصطلحات موضوعية: PATIENT compliance, HETEROZYGOUS familial hypercholesterolemia, LDL cholesterol, STATINS (Cardiovascular agents), TREATMENT effectiveness

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