يعرض 1 - 10 نتائج من 809 نتيجة بحث عن '"Hollak, Carla"', وقت الاستعلام: 0.99s تنقيح النتائج
  1. 1
    دورية أكاديمية
    Perspectives and Update on the Global Shortage of Verteporfin (Visudyne®)

    المؤلفون: Sirks, Marc J., Subhi, YousifAff2, Aff3, Aff4, Rosenberg, NoaAff5, Aff6, Hollak, Carla E. M.Aff5, Aff6, Aff7, Boon, Camiel J. F.Aff1, Aff8, Diederen, Roselie M. H., Yzer, Suzanne, Ossewaarde-van Norel, Jeannette, de Jong-Hesse, Yvonne, Schlingemann, Reinier O.Aff11, Aff12, Moss, Rob J., van Dijk, Elon H. C.Aff8, Aff14, IDs40123024009529_cor12

    المصدر: Ophthalmology and Therapy. 13(7):1821-1831

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  2. 2
    دورية أكاديمية
    Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial.

    المؤلفون: Giugliani, Roberto, Guelbert, Norberto, Hennermann, Julia, Ikezoe, Takayuki, Lidove, Olivier, Mabe, Paulina, Mengel, Eugen, Scarpa, Maurizio, Senates, Ebubekir, Tchan, Michel, Villarrubia, Jesus, Thurberg, Beth, Yarramaneni, Abhimanyu, Armstrong, Nicole, Kim, Yong, Kumar, Monica, Wasserstein, Melissa, Lachmann, Robin, Hollak, Carla, Barbato, Antonio, Gallagher, Renata

    المصدر: Orphanet Journal of Rare Diseases. 18(1)

    مصطلحات موضوعية: Acid sphingomyelinase deficiency, Dose escalation, Lung diffusing capacity, Niemann–Pick type A/B, Niemann–Pick type B, Organomegaly, Recombinant human acid sphingomyelinase, Adult, Humans, Sphingomyelin Phosphodiesterase, Niemann-Pick Disease, Type A, Niemann-Pick Diseases, Recombinant Proteins

    وصف الملف: application/pdf

    URL الوصول: https://escholarship.org/uc/item/3tx7861m

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  3. 3
    دورية أكاديمية
    Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial

    المؤلفون: Wasserstein, Melissa P.Aff1, IDs13023023029830_cor1, Lachmann, Robin, Hollak, Carla, Barbato, Antonio, Gallagher, Renata C., Giugliani, Roberto, Guelbert, Norberto Bernardo, Hennermann, Julia B., Ikezoe, Takayuki, Lidove, Olivier, Mabe, Paulina, Mengel, Eugen, Scarpa, Maurizio, Senates, Ebubekir, Tchan, Michel, Villarrubia, Jesus, Thurberg, Beth L., Yarramaneni, Abhimanyu, Armstrong, Nicole M., Kim, Yong, Kumar, Monica

    المصدر: Orphanet Journal of Rare Diseases. 18(1)

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  4. 4
    دورية أكاديمية
    Consensus clinical management guidelines for acid sphingomyelinase deficiency (Niemann–Pick disease types A, B and A/B)

    المؤلفون: Geberhiwot, TarekegnAff1, Aff2, IDs13023023026866_cor1, Wasserstein, Melissa, Wanninayake, Subadra, Bolton, Shaun Christopher, Dardis, Andrea, Lehman, Anna, Lidove, Olivier, Dawson, Charlotte, Giugliani, Roberto, Imrie, Jackie, Hopkin, Justin, Green, James, de Vicente Corbeira, Daniel, Madathil, Shyam, Mengel, Eugen, Ezgü, Fatih, Pettazzoni, Magali, Sjouke, Barbara, Hollak, Carla, Vanier, Marie T., McGovern, Margaret, Schuchman, Edward

    المصدر: Orphanet Journal of Rare Diseases. 18(1)

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  5. 5
    دورية أكاديمية
    Development of a Biosimilar of Agalsidase Beta for the Treatment of Fabry Disease: Preclinical Evaluation

    المؤلفون: van Kuilenburg, André B. P.Aff1, Aff2, IDs4026802300421x_cor1, Hollak, Carla E. M.Aff2, Aff3, Aff4, Travella, Ana, Jacobs, Melisa, Gentilini, Lucas D., Leen, RenéAff1, Aff2, der Vlugt, Karen M. M. Ghauharali-vanAff1, Aff2, Stet, Femke S. BeersAff1, Aff2, Goorden, Susan M. I.Aff1, Aff2, van der Veen, SanneAff2, Aff3, Aff4, Criscuolo, Marcelo, Papouchado, Mariana

    المصدر: Drugs in R&D. 23(2):141-153

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  6. 6
    دورية أكاديمية
    Illustrating the Financial Consequences of Outcome-Based Payment Models From a Payers Perspective: The Case of Autologous Gene Therapy Atidarsagene Autotemcel (Libmeldy®)

    المؤلفون: Callenbach, Marcelien H.E., Schoenmakers, Daphne, Vreman, Rick A., Vijgen, Sylvia, Timmers, Lonneke, Hollak, Carla E.M., Mantel-Teeuwisse, Aukje K., Goettsch, Wim G.

    المصدر: In Value in Health May 2024

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  7. 7
    دورية أكاديمية
    Glycoprotein non-metastatic protein B (GPNMB) plasma values in patients with chronic visceral acid sphingomyelinase deficiency

    المؤلفون: Eskes, Eline C.B., van der Lienden, Martijn J.C., Sjouke, Barbara, van Vliet, Laura, Brands, Marion M.M.G., Hollak, Carla E.M., Aerts, Johannes M.F.G.

    المصدر: In Molecular Genetics and Metabolism August 2023 139(4)

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  8. 8
    كتاب إلكتروني
    Lipidoses: The Sphingolipidoses, Lysosomal Acid Lipase Deficiency, and Niemann-Pick Type C

    المؤلفون: Hollak, CarlaAff6

    المساهمون: Blau, Nenad, editorAff1, Dionisi Vici, Carlo, editorAff2, Ferreira, Carlos R., editorAff3, Vianey-Saban, Christine, editorAff4, van Karnebeek, Clara D. M., editorAff5

    المصدر: Physician's Guide to the Diagnosis, Treatment, and Follow-Up of Inherited Metabolic Diseases. :1177-1206

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  9. 9
    دورية أكاديمية
    Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

    المؤلفون: Schoenmakers, Daphne H.Aff1, Aff2, Aff3, Beerepoot, ShaniceAff1, Aff4, Aff5, van den Berg, SibrenAff2, Aff3, Adang, Laura, Bley, Annette, Boelens, Jaap-Jan, Fumagalli, Francesca, Goettsch, Wim G.Aff10, Aff11, Grønborg, Sabine, Groeschel, Samuel, van Hasselt, Peter M., Hollak, Carla E. M.Aff2, Aff3, Lindemans, CarolineAff5, Aff15, Mochel, FannyAff16, Aff17, Mol, Peter G. M.Aff18, Aff19, Sevin, CarolineAff20, Aff21, Zerem, AyeletAff22, Aff23, Schöls, LudgerAff24, Aff25, Wolf, Nicole I.Aff1, IDs1302302202189w_cor19

    المصدر: Orphanet Journal of Rare Diseases. 17(1)

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  10. 10
    دورية أكاديمية
    Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study

    المؤلفون: Eskes, Eline C. B.Aff1, Aff2, Beishuizen, Cathrien R. L., Corazolla, Eleonore M.Aff1, Aff2, Aff4, van Middelaar, Tessa, Brands, Marion M. M. G.Aff2, Aff6, Dekker, Hanka, van de Mheen, Erica, Langeveld, MirjamAff1, Aff2, Hollak, Carla E. M.Aff1, Aff2, Sjouke, BarbaraAff1, Aff2, IDs1302302202543y_cor10

    المصدر: Orphanet Journal of Rare Diseases. 17(1)

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