Showing 1 - 8 results of 8 for search 'Manisha Balwani', query time: 0.04s Refine Results
  1. 1
    Porphyrias in the Age of Targeted Therapies

    Porphyrias in the Age of Targeted Therapies by Angelika L. Erwin, Manisha Balwani

    Published 2021-09-01
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  2. 2
    P013: Congenital erythropoietic porphyria: Variable age of onset of porphyrinuria, an indicator of disease severity and correlation with genotype

    P013: Congenital erythropoietic porphyria: Variable age of onset of porphyrinuria, an indicator of disease severity and correlation with genotype by Angelika Erwin, Manoj Agarwala, Hetanshi Naik, Allison Schreiber, Manisha Balwani, Robert Desnick

    Published 2023-01-01
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  3. 3
    Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study

    Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study by Eliane Sardh, Manisha Balwani, David C. Rees, Karl E. Anderson, Gang Jia, Marianne T. Sweetser, Bruce Wang

    Published 2024-10-01
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  4. 4
    P1588: LONG-TERM REAL-WORLD EFFECTIVENESS OF ELIGLUSTAT IN TREATMENT-NAÏVE AND SWITCH PATIENTS ENROLLED IN THE INTERNATIONAL COLLABORATIVE GAUCHER GROUP (ICGG) GAUCHER REGISTRY

    P1588: LONG-TERM REAL-WORLD EFFECTIVENESS OF ELIGLUSTAT IN TREATMENT-NAÏVE AND SWITCH PATIENTS ENROLLED IN THE INTERNATIONAL COLLABORATIVE GAUCHER GROUP (ICGG) GAUCHER REGISTRY by Pramod Mistry, Manisha Balwani, Joel Charrow, Jeremy Lorber, Claus Niederau, Jenny Carwile, Sefika Uslu, Priya S. Kishnani

    Published 2023-08-01
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  5. 5
    Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study

    Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study by Bruce Wang, Paolo Ventura, Kei-ichiro Takase, Manish Thapar, David Cassiman, Ilja Kubisch, Shangbin Liu, Marianne T. Sweetser, Manisha Balwani

    Published 2022-08-01
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  6. 6
    A pilot study of oral iron therapy in erythropoietic protoporphyria and X-linked protoporphyria

    A pilot study of oral iron therapy in erythropoietic protoporphyria and X-linked protoporphyria by Manisha Balwani, Hetanshi Naik, Jessica R. Overbey, Herbert L. Bonkovsky, D. Montgomery Bissell, Bruce Wang, John D. Phillips, Robert J. Desnick, Karl E. Anderson

    Published 2022-12-01
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  7. 7
    PB2562: STUDY DESIGN OF THE AURORA TRIAL: A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL OF BITOPERTIN IN ERYTHROPOIETIC PROTOPORPHYRIA

    PB2562: STUDY DESIGN OF THE AURORA TRIAL: A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL OF BITOPERTIN IN ERYTHROPOIETIC PROTOPORPHYRIA by Cynthia Levy, Karl E. Anderson, Manisha Balwani, Herbert L. Bonkovsky, Amy Dickey, Siobán Keel, Brendan Mcguire, Roy Smith, Manish Thapar, Bruce Wang, William Savage

    Published 2023-08-01
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  8. 8
    Loss-of-Function Ferrochelatase and Gain-of-Function Erythroid-Specific 5-Aminolevulinate Synthase Mutations Causing Erythropoietic Protoporphyria and X-Linked Protoporphyria in North American Patients Reveal Novel Mutations and a High Prevalence of X-Linked Protoporphyria

    Loss-of-Function Ferrochelatase and Gain-of-Function Erythroid-Specific 5-Aminolevulinate Synthase Mutations Causing Erythropoietic Protoporphyria and X-Linked Protoporphyria in No... by Manisha Balwani, Dana Doheny, David F. Bishop, Irina Nazarenko, Makiko Yasuda, Harry A. Dailey, Karl E. Anderson, D. Montgomery Bissell, Joseph Bloomer, Herbert L. Bonkovsky, John D. Phillips, Lawrence Liu, Robert J. Desnick, The Porphyrias Consortium of the National Institutes of Health Rare Diseases Clinical Research Network

    Published 2013-01-01
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