The effect of the COVID-19 pandemic on US Food and Drug Administration-funded clinical trials and natural history studies for rare diseases

por Miller, Kathleen L., Miller Needleman, Katherine I.
Publicado 2023

Precision Medicines’ Impact on Orphan Drug Designation

por Mueller, Christine M., Rao, Gayatri R., Miller Needleman, Katherine I.
Publicado 2019

FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development

por Miller, Kathleen L., Mueller, Christine, Liu, Gumei, Miller Needleman, Katherine I., Maynard, Janet
Publicado 2020

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