Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Authors: 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

Source: Frontiers in Neurology; 2025, p1-15, 15p

Subject Terms: GLYCOGEN storage disease type II, ENZYME replacement therapy, LYSOSOMAL storage diseases, PATIENT experience, ENZYME activation, GLYCANS

Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Authors: Byrne, Barry J., Parenti, Giancarlo, Schoser, Benedikt, van der Ploeg, Ans T., Hung Do, Fox, Brian, Goldman, Mitchell, Johnson, Franklin K., Jia Kang, Mehta, Nickita, Mondick, John, Sheikh, M. Osman, Das, Sheela Sitaraman, Tuske, Steven, Brudvig, Jon, Weimer, Jill M., Mozaffar, Tahseen

Source: Frontiers in Neurology; 2024, p1-15, 15p

Subject Terms: GLYCOGEN storage disease type II, ENZYME replacement therapy, LYSOSOMAL storage diseases, PATIENT experience, ENZYME activation, METABOLIC disorders, GLYCANS

Population pharmacokinetics of total and unbound valemetostat and platelet dynamics in healthy volunteers and patients with non‐Hodgkin lymphoma.

Authors: Fukae, Masato, Baron, Kyle, Tachibana, Masaya, Mondick, John, Shimizu, Takako

Source: CPT: Pharmacometrics & Systems Pharmacology; Oct2024, Vol. 13 Issue 10, p1641-1654, 14p

Subject Terms: PLATELET count, TERMINATION of treatment, BLOOD platelets, HOCKEY, CLINICAL trials

Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Authors: 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

Source: Frontiers in Neurology; 2025, p1-3, 3p

Subject Terms: GLYCOGEN storage disease type II, LYSOSOMAL storage diseases, ENZYME replacement therapy, TREATMENT effectiveness, SCHOLARLY periodical corrections

Population pharmacokinetics of adalimumab biosimilar adalimumab‐adbm and reference product in healthy subjects and patients with rheumatoid arthritis to assess pharmacokinetic similarity

Authors: Kang, Jia, Eudy‐Byrne, Rena J., Mondick, John, Knebel, William, Jayadeva, Girish, Liesenfeld, Karl‐Heinz

Source: British journal of clinical pharmacology. 86(11):2274-2285

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100108624334.0x000001&indx=1&recIds=ETOCvdc_100108624334.0x000001

Latent process model of the 6-minute walk test in Duchenne muscular dystrophy

Authors: Lennie, Janelle L., Mondick, John T., Gastonguay, Marc R.

Source: Journal of pharmacokinetics and pharmacodynamics. 47(1):91-104

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100164189118.0x000001&indx=1&recIds=ETOCvdc_100164189118.0x000001

Bayesian modeling and simulation to inform rare disease drug development early decision-making: Application to Duchenne muscular dystrophy.

Authors: Lennie, Janelle L.^1,2 (AUTHOR) janellelennie@gmail.com, Mondick, John T.² (AUTHOR), Gastonguay, Marc R.^1,2 (AUTHOR)

Source: PLoS ONE. 4/28/2022, Vol. 17 Issue 4, p1-14. 14p.

Subject Terms: *DUCHENNE muscular dystrophy, *RARE diseases, *DRUG development, *BAYESIAN analysis, *SIMULATION methods & models

Population Pharmacokinetic– Pharmacodynamic Analysis to Characterize the Effect of Empagliflozin on Renal Glucose Threshold in Patients With Type 1 Diabetes Mellitus

Authors: Mondick, John, Riggs, Matthew, Kaspers, Stefan, Soleymanlou, Nima, Marquard, Jan, Nock, Valerie

Source: Journal of clinical pharmacology. 58(5):640-649

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100108119794.0x000001&indx=1&recIds=ETOCvdc_100108119794.0x000001

Bayesian estimation in NONMEM.

Authors: Johnston, Curtis K., Waterhouse, Timothy, Wiens, Matthew, Mondick, John, French, Jonathan, Gillespie, William R.

Source: CPT: Pharmacometrics & Systems Pharmacology; Feb2024, Vol. 13 Issue 2, p192-207, 16p

Subject Terms: DRUG development, PHARMACOKINETICS

Malabsorption blood test: Assessing fat absorption in patients with cystic fibrosis and pancreatic insufficiency

Authors: Mascarenhas, Maria R., Mondick, John, Barrett, Jeffrey S., Wilson, Martha, Stallings, Virginia A., Schall, Joan I.

Source: Journal of clinical pharmacology. 55(8):854-865

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100150094847.0x000001&indx=1&recIds=ETOCvdc_100150094847.0x000001

3.1 Population Pharmacokinetic-Pharmacodynamic Modeling of Variable Wear Times for a Dextroamphetamine Transdermal System

Authors: Meeves, Suzanne, Castelli, Mariacristina, Komaroff, Marina, Balakrishnan, Kanan, Baron, Kyle T., Mondick, John T., Faraone, Stephen V., Mattingly, Gregory W.

Source: Journal of the American Academy of Child & Adolescent Psychiatry. 61(10):S228-S228

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100173820359.0x000001&indx=1&recIds=ETOCvdc_100173820359.0x000001

Immunogenicity of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease (LOPD): A phase III, randomized study (PROPEL)

Authors: 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

Source: Molecular genetics and metabolism. 135(2):S30-S30

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100155636671.0x000001&indx=1&recIds=ETOCvdc_100155636671.0x000001

Plasma total GAA protein PK profiles differ between cipaglucosidase alfa/miglustat and alglucosidase alfa

Authors: Johnson, Franklin K., Kang, Jia, Mondick, John, Sitaraman, Sheela, Wasfi, Yasmine, Goldman, Mitchell, Castelli, Jeff, Do, Hung

Source: Molecular genetics and metabolism. 135(2):S63-S63

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100154953117.0x000001&indx=1&recIds=ETOCvdc_100154953117.0x000001

Integration of modeling and simulation into hospital-based decision support systems guiding pediatric pharmacotherapy

Authors: Vijayakumar Kalpana, Narayan Mahesh, Mondick John T, Barrett Jeffrey S, Vijayakumar Sundararajan

Source: BMC Medical Informatics and Decision Making, Vol 8, Iss 1, p 6 (2008)

Subject Terms: Computer applications to medicine. Medical informatics, R858-859.7

File Description: electronic resource

Relation: http://www.biomedcentral.com/1472-6947/8/6; https://doaj.org/toc/1472-6947

Access URL: https://doaj.org/article/def9a3ef3d1e4b4c8023cb6e6fb394a9

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