Search Results - "Das, Sheela Sitaraman"

Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease by Byrne, Barry J., Parenti, Giancarlo, Schoser, Benedikt, van der Ploeg, Ans T., Do, Hung, Fox, Brian, Goldman, Mitchell, Johnson, Franklin K., Kang, Jia, Mehta, Nickita, Mondick, John, Sheikh, M. Osman, Sitaraman Das, Sheela, Tuske, Steven, Brudvig, Jon, Weimer, Jill M., Mozaffar, Tahseen

“…Enzyme replacement therapy (ERT) is the only approved disease-modifying treatment modality for Pompe disease, a rare, inherited metabolic disorder caused by a…”
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Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data by Kishnani, Priya S., Shohet, Simon, Raza, Syed, Hummel, Noemi, Castelli, Jeffrey P., Sitaraman Das, Sheela, Jiang, Heng, Kopiec, Agnieszka, Keyzor, Ian, Hahn, Andreas

“…Background The construct validity and interpretation of the Patient-Reported Outcome Measurement Information System (PROMIS ® ) Physical Function short form…”
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P021: Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory and non-ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02) by Kishnani, Priya, Schoser, Benedikt, Bratkovic, Drago, Clemens, Paula, Goker-Alpan, Ozlem, Ming, Xue, Roberts, Mark, Vorgerd, Matthias, Sivakumar, Kumaraswamy, van der Ploeg, Ans, Goldman, Mitchell, Wright, Jacquelyn, Holdbrook, Fred, Jain, Vipul, Das, Sheela Sitaraman, Wasfi, Yasmine, Mozaffar, Tahseen, Byrne, Barry

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Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL by Mozaffar, Tahseen, Bratkovic, Drago, Byrne, Barry J., Claeys, Kristl G., Diaz-Manera, Jordi, Dimachkie, Mazen M., Kishnani, Priya S., Kushlaf, Hani, Roberts, Mark E., Toscano, Antonio, Castelli, Jeffrey, Holdbrook, Fred K., Das, Sheela Sitaraman, Wasfi, Yasmine, Schoser, Benedikt

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Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02) by Byrne, Barry J., Schoser, Benedikt, Kishnani, Priya S., Bratkovic, Drago, Clemens, Paula R., Goker-Alpan, Ozlem, Ming, Xue, Roberts, Mark, Vorgerd, Matthias, Sivakumar, Kumaraswamy, van der Ploeg, Ans T., Goldman, Mitchell, Wright, Jacquelyn, Holdbrook, Fred, Jain, Vipul, Benjamin, Elfrida R., Johnson, Franklin, Das, Sheela Sitaraman, Wasfi, Yasmine, Mozaffar, Tahseen

“…Cipaglucosidase alfa plus miglustat (cipa + mig) is a novel, two-component therapy for Pompe disease. We report data from the Phase I/II ATB200-02 study for up…”
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Immunogenicity of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease (LOPD): A phase III, randomized study (PROPEL) by Chien, Yin-Hsiu, Benjamin, Elfrida, Schoser, Benedikt, Kishnani, Priya, Mozaffar, Tahseen, Díaz-Manera, Jordi, Johnson, Franklin, Das, Sheela Sitaraman, Nair, Anju, Williams, Hadis, Anderson, Eric, Mondick, John T., Sileno, Anthony

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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07) by Schoser, Benedikt, Kishnani, Priya S., Bratkovic, Drago, Byrne, Barry J., Claeys, Kristl G., Díaz-Manera, Jordi, Laforêt, Pascal, Roberts, Mark, Toscano, Antonio, van der Ploeg, Ans T., Castelli, Jeff, Goldman, Mitchell, Holdbrook, Fred, Sitaraman Das, Sheela, Wasfi, Yasmine, Mozaffar, Tahseen

“…The phase III double-blind PROPEL study compared the novel two-component therapy cipaglucosidase alfa + miglustat (cipa + mig) with alglucosidase alfa +…”
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