Hurricane risk assessment in a multi-hazard context for Dominica in the Caribbean

por Sammonds, Peter, Alam, Akhtar, Day, Simon, Stavrianaki, Katerina, Kelman, Ilan
Publicado 2023

Clinical trials in rare diseases: a review of practice

por Bell, Stuart, Williamson, Paula, Day, Simon, Wheatley, Keith, Whitehead, John, Smith, Catrin Tudur
Publicado 2013

Determination of the optimal sample size for a clinical trial accounting for the population size

por Stallard, Nigel, Miller, Frank, Day, Simon, Hee, Siew Wan, Madan, Jason, Zohar, Sarah, Posch, Martin
Publicado 2016

Sharing clinical trial data on patient level: Opportunities and challenges

por Koenig, Franz, Slattery, Jim, Groves, Trish, Lang, Thomas, Benjamini, Yoav, Day, Simon, Bauer, Peter, Posch, Martin
Publicado 2015

Decision-theoretic designs for small trials and pilot studies: A review

por Hee, Siew Wan, Hamborg, Thomas, Day, Simon, Madan, Jason, Miller, Frank, Posch, Martin, Zohar, Sarah, Stallard, Nigel
Publicado 2015

Value of information methods to design a clinical trial in a small population to optimise a health economic utility function

por Pearce, Michael, Hee, Siew Wan, Madan, Jason, Posch, Martin, Day, Simon, Miller, Frank, Zohar, Sarah, Stallard, Nigel
Publicado 2018

Utilising benefit-risk assessments within clinical trials—a protocol for the BRAINS project

por Totton, Nikki, Julious, Steven, Hughes, Dyfrig, Cook, Jonathan, Biggs, Katie, Coates, Lizzie, Cook, Andrew, Hewitt, Catherine, Day, Simon
Publicado 2021

Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials.gov

por Hee, Siew Wan, Willis, Adrian, Tudur Smith, Catrin, Day, Simon, Miller, Frank, Madan, Jason, Posch, Martin, Zohar, Sarah, Stallard, Nigel
Publicado 2017

Recommendations for the design of small population clinical trials

por Day, Simon, Jonker, Anneliene Hechtelt, Lau, Lilian Pek Lian, Hilgers, Ralf-Dieter, Irony, Ilan, Larsson, Kristina, Roes, Kit CB, Stallard, Nigel
Publicado 2018

c4c: Paediatric pharmacovigilance: Methodological considerations in research and development of medicines for children – A c4c expert group white paper

por Aurich, Beate, Apele‐Freimane, Dina, Banaschewski, Tobias, Chouchana, Laurent, Day, Simon, Kaguelidou, Florentia, Kelly, Lauren E., Kindblom, Jenny M., Neubert, Antje, Wong, Ian C. K.
Publicado 2021

Recent advances in methodology for clinical trials in small populations: the InSPiRe project

por Friede, Tim, Posch, Martin, Zohar, Sarah, Alberti, Corinne, Benda, Norbert, Comets, Emmanuelle, Day, Simon, Dmitrienko, Alex, Graf, Alexandra, Günhan, Burak Kürsad, Hee, Siew Wan, Lentz, Frederike, Madan, Jason, Miller, Frank, Ondra, Thomas, Pearce, Michael, Röver, Christian, Toumazi, Artemis, Unkel, Steffen, Ursino, Moreno, Wassmer, Gernot, Stallard, Nigel
Publicado 2018

Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study

por Mengel, Eugen, Bembi, Bruno, del Toro, Mireia, Deodato, Federica, Gautschi, Matthias, Grunewald, Stephanie, Grønborg, Sabine, Héron, Bénédicte, Maier, Esther M., Roubertie, Agathe, Santra, Saikat, Tylki-Szymanska, Anna, Day, Simon, Symonds, Tara, Hudgens, Stacie, Patterson, Marc C., Guldberg, Christina, Ingemann, Linda, Petersen, Nikolaj H. T., Kirkegaard, Thomas, í Dali, Christine
Publicado 2020

Correction to: Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study

por Mengel, Eugen, Bembi, Bruno, del Toro, Mireia, Deodato, Federica, Gautschi, Matthias, Grunewald, Stephanie, Grønborg, Sabine, Héron, Bénédicte, Maier, Esther M., Roubertie, Agathe, Santra, Saikat, Tylki-Szymanska, Anna, Day, Simon, Symonds, Tara, Hudgens, Stacie, Patterson, Marc C., Guldberg, Christina, Ingemann, Linda, Petersen, Nikolaj H. T., Kirkegaard, Thomas, Dali, Christine í
Publicado 2021

Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment...

por Mengel, Eugen, Patterson, Marc C., Da Riol, Rosalia M., Del Toro, Mireia, Deodato, Federica, Gautschi, Matthias, Grunewald, Stephanie, Grønborg, Sabine, Harmatz, Paul, Héron, Bénédicte, Maier, Esther M., Roubertie, Agathe, Santra, Saikat, Tylki‐Szymanska, Anna, Day, Simon, Andreasen, Anne Katrine, Geist, Marie Aavang, Havnsøe Torp Petersen, Nikolaj, Ingemann, Linda, Hansen, Thomas, Blaettler, Thomas, Kirkegaard, Thomas, í Dali, Christine
Publicado 2021

8th European Conference on Rare Diseases & Orphan Products (ECRD 2016): Edinburgh, UK. 26-28 May 2016

por Schlander, Michael, Holm, Søren, Nord, Erik, Richardson, Jeff, Garattini, Silvio, Kolominsky-Rabas, Peter, Marshall, Deborah, Persson, Ulf, Postma, Maarten, Simoens, Steven, de Solà Morales, Oriol, Tolley, Keith, Toumi, Mondher, Telser, Harry, Bonham, James R, Hintner, Helmut, Diem, Anja, Laimer, Martin, Hébert, Réjean, Dasgupta, Nabarun, Pierce, Carrie E., Jordan, Melissa, Bori, Barbara, Fors, Mohanad, Prazakova, Emilie, Day, Simon, Croce, Thomas J., Fransson, Jonas, Wood, Philip, Lauridsen, Anne-Grethe, Higgs, Joanne, Aleksovska, Vesna Stojmirova, Olsen, Christina, Head, Ritchie, Asero, Antonio, Papa, Vincenzo, van Kan, Christa, Favennec, Loic, Venturella, Silvana, Salvador, Michela, Krol, Alan, Nielsen, Stephanie J., Holm, Birthe B., Lewi, Daniel, Durão, Patricia, Band, Heather, West, Andrea, Hammann, Marinda J. A., Effing-Boele, Marije C., Dekker, Hanka K., Hunter, Amy, Simpson, Amy, Liu, Gumei, Needleman, Katherine, Lewis, Debra, Rao, Gayatri, Simpson, Amy, Hunter, Amy, Whitaker, Martin J., Castro, Raquel
Publicado 2016