Showing 1 - 7 results of 7 for search 'Paul Harmatz', query time: 0.01s Refine Results
  1. 1
    Enzyme replacement therapy with galsulfase for mucopolysaccharidosis VI: clinical facts and figures

    Enzyme replacement therapy with galsulfase for mucopolysaccharidosis VI: clinical facts and figures by Paul Harmatz

    Published 2010-10-01
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  2. 2
    Current and new therapies for mucopolysaccharidoses

    Current and new therapies for mucopolysaccharidoses by Monica Penon-Portmann, David R. Blair, Paul Harmatz

    Published 2023-02-01
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  3. 3
    P833: Persistent clenched hands with or without adducted thumbs: A fetal ultrasound finding in neuronopathic Gaucher disease

    P833: Persistent clenched hands with or without adducted thumbs: A fetal ultrasound finding in neuronopathic Gaucher disease by Billie Lianoglou, Marjorie Treadwell, Teresa Sparks, Juan Gonzalez-Velez, Dawn Gano, Akos Herzeg, Emma Canepa, Paul Harmatz, Irene Chang, Tippi MacKenzie

    Published 2025-01-01
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  4. 4
    Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey

    Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey by Joseph Muenzer, Hernan Amartino, Roberto Giugliani, Paul Harmatz, Shuan-Pei Lin, Bianca Link, David Molter, Uma Ramaswami, Maurizio Scarpa, Jaco Botha, Jennifer Audi, Barbara K. Burton

    Published 2025-07-01
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  5. 5
    P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study

    P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study by Barbara Burton, Joseph Muenzer, Paul Harmatz, Deepa Rajan, Simon Jones, Johanna van den Hout, John Mitchell, Matthew Troyer, Natalie Engmann, Rupa Caprihan, Akhil Bhalla, Imanol Zubizarreta, Arlette Weichert, Peter Chin, Carole Ho

    Published 2025-01-01
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  6. 6
    O13: Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study

    O13: Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study by Ravi Savarirayan, Elena Muslimova, Josep Bergua, Paul Arundel, Jean Salles, Vrinda Saraff, Borja Delgado, Antonio Leiva-Gea, Helen McDevitt, Marc Nicolino, Massimiliano Rossi, Maria Salcedo, Valerie Cormier-Daire, Mars Skael, Peter Kannu, John Phillips, Howard Saal, Paul Harmatz, Toby Candler, Richard Weng, Yun Bai, Supriya Raj, Julie Hoover-Fong, Melita Irving, Daniela Rogoff

    Published 2025-01-01
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  7. 7
    Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale

    Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severi... by Eugen Mengel, Marc C. Patterson, Rosalia M. Da Riol, Mireia Del Toro, Federica Deodato, Matthias Gautschi, Stephanie Grunewald, Sabine Weller Grønborg, Paul Harmatz, Julia B. Hennermann, Bénédicte Héron, Esther M. Maier, Agathe Roubertie, Saikat Santra, Anna Tylki-Szymanska, Lisa LaGorio, Elizabeth Berry-Kravis, Forbes D. Porter, Beth Solomon, Louise Himmelstrup, Travis Mickle, Sven Guenther, Christine í Dali

    Published 2025-06-01
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