Zobrazeno 1 - 10
of 47
pro vyhledávání: '"Kathleen L. Miller"'
Autor:
Catherine Mease, Kathleen L. Miller, Lewis J. Fermaglich, Jeanine Best, Gumei Liu, Erika Torjusen
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-9 (2024)
Abstract Background The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program was enacted in 2012 to support the development of new products for children. Prior to requesting a voucher, applicants can request RPD designation, which confi
Externí odkaz:
https://doaj.org/article/aca57dc24c7544cd812cf6066282b494
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-8 (2023)
Abstract Background Rare diseases affect more than 30 million Americans. The passage of the Orphan Drug Act (ODA) in the United States in 1983 represented a launching point for a rare disease drug development revolution for these patients. Financial
Externí odkaz:
https://doaj.org/article/59aaeb22405849489c5cffc34bad6ba8
Autor:
Catherine Mease, Kathleen L. Miller, Lewis J. Fermaglich, Jeanine Best, Gumei Liu, Erika Torjusen
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-1 (2024)
Externí odkaz:
https://doaj.org/article/a768d1046fc34402a4219fc4eeee499f
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-10 (2021)
Abstract Background Orphan drug designations are a useful proxy to investigate trends in rare disease drug development. Drug developers must receive a designation before they are eligible for the economic incentives of the Orphan Drug Act in the Unit
Externí odkaz:
https://doaj.org/article/f738e0c4f7b64ba48415aa6d9b1aac59
Autor:
Kathleen L. Miller, Christine Mueller, Gumei Liu, Katherine I. Miller Needleman, Janet Maynard
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-8 (2020)
Abstract Background The Office of Orphan Products Development (OOPD) of the United States (U.S.) Food and Drug Administration (FDA) has awarded over 700 grants to conduct clinical trials of medicals products for rare diseases since 1983, leading to o
Externí odkaz:
https://doaj.org/article/6c5574fc173f4c58941d2f5d29680c63
Autor:
Kathleen L. Miller, Michael Lanthier
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 13, Iss 1, Pp 1-8 (2018)
Abstract Background The Orphan Drug Act was enacted in 1983 to encourage the development of drugs for rare diseases. Previous research has attempted to examine the impact of the Act by assessing either the number of orphan designations that have been
Externí odkaz:
https://doaj.org/article/999b727b54c24e4997088baf5cabb9b0
Autor:
Keith H. K. Wong, Shannon N. Tessier, David T. Miyamoto, Kathleen L. Miller, Lauren D. Bookstaver, Thomas R. Carey, Cleo J. Stannard, Vishal Thapar, Eric C. Tai, Kevin D. Vo, Erin S. Emmons, Haley M. Pleskow, Rebecca D. Sandlin, Lecia V. Sequist, David T. Ting, Daniel A. Haber, Shyamala Maheswaran, Shannon L. Stott, Mehmet Toner
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-11 (2017)
The current FDA-approved whole blood stabilization method for circulating tumor cell (CTC) isolation suffers from RNA degradation. Here the authors combine hypothermic preservation and antiplatelet strategies to stabilize whole blood up to 72 h witho
Externí odkaz:
https://doaj.org/article/2cb65508990f455f91b7d509541cd82f
Autor:
Rebecca D. Sandlin, Keith H. K. Wong, Leo Boneschansker, Thomas R. Carey, Kathleen L. Miller, Gregory Rose, Daniel A. Haber, Shyamala Maheswaran, Daniel Irimia, Shannon L. Stott, Mehmet Toner
Publikováno v:
Scientific Reports, Vol 7, Iss 1, Pp 1-11 (2017)
Abstract The deterioration of whole blood ex vivo represents a logistical hurdle in clinical and research settings. Here, a cocktail preservative is described that stabilizes leukocyte viability and erythrocyte morphology in whole blood under ambient
Externí odkaz:
https://doaj.org/article/c57942a3dc7846bba76a9debd6c2565f
Autor:
Kathleen L. Miller
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 12, Iss 1, Pp 1-6 (2017)
Abstract Background The Orphan Drug Act is an important piece of legislation that uses financial incentives to encourage the development of drugs that treat rare diseases. This analysis studies the effects of a portion of the Orphan Drug Act, the orp
Externí odkaz:
https://doaj.org/article/cb1e14e2653e42bd83fa9917f61d7e15
Publikováno v:
Expert Opin Orphan Drugs
BACKGROUND: The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding of rare disease drug development, as
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::177cd013811221a44ae9aa40045eaf8b
https://doi.org/10.1080/21678707.2021.2047021
https://doi.org/10.1080/21678707.2021.2047021