From scientific discovery to treatments for rare diseases - the view from the National Center for Advancing Translational Sciences - Office of Rare Diseases Research
Kaufmann, Petra, Pariser, Anne R, Austin, Christopher
Published in Orphanet journal of rare diseases (06.11.2018)
Published in Orphanet journal of rare diseases (06.11.2018)
Get full text
Journal Article
The IDeaS initiative: pilot study to assess the impact of rare diseases on patients and healthcare systems
Tisdale, Ainslie, Cutillo, Christine M, Nathan, Ramaa, Russo, Pierantonio, Laraway, Bryan, Haendel, Melissa, Nowak, Douglas, Hasche, Cindy, Chan, Chun-Hung, Griese, Emily, Dawkins, Hugh, Shukla, Oodaye, Pearce, David A, Rutter, Joni L, Pariser, Anne R
Published in Orphanet journal of rare diseases (22.10.2021)
Published in Orphanet journal of rare diseases (22.10.2021)
Get full text
Journal Article
Operational description of rare diseases: a reference to improve the recognition and visibility of rare diseases
Wang, Chiuhui Mary, Whiting, Amy Heagle, Rath, Ana, Anido, Roberta, Ardigò, Diego, Baynam, Gareth, Dawkins, Hugh, Hamosh, Ada, Le Cam, Yann, Malherbe, Helen, Molster, Caron M, Monaco, Lucia, Padilla, Carmencita D, Pariser, Anne R, Robinson, Peter N, Rodwell, Charlotte, Schaefer, Franz, Weber, Stefanie, Macchia, Flaminia
Published in Orphanet journal of rare diseases (11.09.2024)
Published in Orphanet journal of rare diseases (11.09.2024)
Get full text
Journal Article
Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force
Nabbout, Rima, Zanello, Galliano, Baker, Dixie, Black, Lora, Brambilla, Isabella, Buske, Orion J, Conklin, Laurie S, Davies, Elin Haf, Julkowska, Daria, Kim, Yeonju, Klopstock, Thomas, Nakamura, Harumasa, Nielsen, Kim G, Pariser, Anne R, Pastor, Jose Carlos, Scarpa, Maurizio, Smith, Maureen, Taruscio, Domenica, Groft, Stephen
Published in Orphanet journal of rare diseases (09.05.2023)
Published in Orphanet journal of rare diseases (09.05.2023)
Get full text
Journal Article
Cross-sector sponsorship of research in eosinophilic esophagitis: A collaborative model for rational drug development in rare diseases
Fiorentino, Robert, MD, Liu, Gumei, MD, PhD, Pariser, Anne R., MD, Mulberg, Andrew E., MD
Published in Journal of allergy and clinical immunology (01.09.2012)
Published in Journal of allergy and clinical immunology (01.09.2012)
Get full text
Journal Article
Representation of Women and Minorities in Clinical Trials for New Molecular Entities and Original Therapeutic Biologics Approved by FDA CDER from 2013 to 2015
Chen, Alice, Wright, Hilary, Itana, Hawi, Elahi, Merina, Igun, Ayomide, Soon, Guoxing, Pariser, Anne R, Fadiran, Emmanuel O
Published in Journal of women's health (Larchmont, N.Y. 2002) (01.04.2018)
Published in Journal of women's health (Larchmont, N.Y. 2002) (01.04.2018)
Get more information
Journal Article
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience
Lomash, Richa Madan, Shchelochkov, Oleg, Chandler, Randy J, Venditti, Charles P, Pariser, Anne R, Ottinger, Elizabeth A
Published in Human gene therapy (01.03.2023)
Published in Human gene therapy (01.03.2023)
Get more information
Journal Article
A role for plasma cell targeting agents in immune tolerance induction in autoimmune disease and antibody responses to therapeutic proteins
Rosenberg, A.S, Pariser, A.R, Diamond, B, Yao, L, Turka, L.A, Lacana, E, Kishnani, P.S
Published in Clinical immunology (Orlando, Fla.) (01.04.2016)
Published in Clinical immunology (Orlando, Fla.) (01.04.2016)
Get full text
Journal Article
Ketoconazole 2% shampoo in the treatment of tinea versicolor: A multicenter, randomized, double-blind, placebo-controlled trial
Lange, David S., Richards, Henry M., Guarnieri, Joseph, Humeniuk, John M., Savin, Ronald C., Reyes, Blas A., Hickman, Janet, Pariser, David M., Pariser, Robert J., Sherertz, Elizabeth F., Grossman, Rachel M., Gisoldi, Elvira M., Klausner, Mark A.
Published in Journal of the American Academy of Dermatology (01.12.1998)
Published in Journal of the American Academy of Dermatology (01.12.1998)
Get full text
Journal Article
Clinical pharmacology as a cornerstone of orphan drug development
Bashaw, Edward D, Huang, Shiew-Mei, Coté, Timothy R, Pariser, Anne R, Garnett, Christine E, Burckart, Gilbert, Zhang, Lei, Men, Angela Y, Le, Christine D, Charlab, Rosane, Gobburu, Jogaro V, Lesko, Lawrence J
Published in Nature reviews. Drug discovery (01.11.2011)
Published in Nature reviews. Drug discovery (01.11.2011)
Get full text
Journal Article
A double-blind, placebo-controlled study of topical tetracaine in the treatment of herpes labialis
Kaminester, Lewis H., Pariser, Robert J., Pariser, David M., Weiss, Jonathan S., Shavin, Joel S., Landsman, Larry, Haines, Harold G., Osborne, David W.
Published in Journal of the American Academy of Dermatology (01.12.1999)
Published in Journal of the American Academy of Dermatology (01.12.1999)
Get full text
Journal Article
Once-weekly fluconazole (450 mg) for 4, 6, or 9 months of treatment for distal subungual onychomycosis of the toenail
Ling, Mark R., Swinyer, Leonard J., Jarratt, Michael Taylor, Falo, Louis, Monroe, Eugene W., Tharp, Michael, Kalivas, James, Weinstein, Gerald D., Asarch, Richard G., Drake, Lynn, Martin, Ann G., Leyden, James J., Cook, Joel, Pariser, David M., Pariser, Robert, Thiers, Bruce H., Lebwohl, Mark G., Babel, Dennis, Stewart, Daniel M., Eaglstein, William H., Falanga, Vincent, Katz, H.Irving, Bergfeld, Wilma F., Hanifin, Jon M., Kang, Sewon, McDonald, Charles J., Muglia, Jennie, Goffe, Bernard S., Young, Marvin R.
Published in Journal of the American Academy of Dermatology (01.06.1998)
Published in Journal of the American Academy of Dermatology (01.06.1998)
Get full text
Journal Article