Zobrazeno 1 - 10
of 29
pro vyhledávání: '"Anne R. Pariser"'
Autor:
Rima Nabbout, Galliano Zanello, Dixie Baker, Lora Black, Isabella Brambilla, Orion J. Buske, Laurie S. Conklin, Elin Haf Davies, Daria Julkowska, Yeonju Kim, Thomas Klopstock, Harumasa Nakamura, Kim G. Nielsen, Anne R. Pariser, Jose Carlos Pastor, Maurizio Scarpa, Maureen Smith, Domenica Taruscio, Stephen Groft
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-10 (2023)
Abstract Background Many patients with rare diseases are still lacking a timely diagnosis and approved therapies for their condition despite the tremendous efforts of the research community, biopharmaceutical, medical device industries, and patient s
Externí odkaz:
https://doaj.org/article/c9ca331354bc45bb934c597b9e06fa26
Autor:
Ainslie Tisdale, Christine M. Cutillo, Ramaa Nathan, Pierantonio Russo, Bryan Laraway, Melissa Haendel, Douglas Nowak, Cindy Hasche, Chun-Hung Chan, Emily Griese, Hugh Dawkins, Oodaye Shukla, David A. Pearce, Joni L. Rutter, Anne R. Pariser
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-18 (2021)
Abstract Background Rare diseases (RD) are a diverse collection of more than 7–10,000 different disorders, most of which affect a small number of people per disease. Because of their rarity and fragmentation of patients across thousands of differen
Externí odkaz:
https://doaj.org/article/bd3dbd832fd6404ca21a136660f71696
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 13, Iss 1, Pp 1-8 (2018)
Abstract We now live in a time of unprecedented opportunities to turn scientific discoveries into better treatments for the estimated 30 million people in the US living with rare diseases. Despite these scientific advances, more than 90% of rare dise
Externí odkaz:
https://doaj.org/article/ed401ffebe524ad7acc307ccd5209557
Autor:
Hawi Itana, Merina Elahi, Emmanuel O. Fadiran, Hilary Wright, Guoxing Soon, Alice Chen, Ayomide Igun, Anne R. Pariser
Publikováno v:
Journal of Women's Health. 27:418-429
Background: The U.S. Food and Drug Administration (FDA) has made efforts to encourage adequate assessment of women, racial/ethnic minorities, and geriatric participants in clinical trials through regulations and guidance documents. This study surveye
Autor:
Yow Ming Wang, Barbara K. Burton, Melissa Hogan, Jeffrey A. Bluestone, Stephen Holland, Anne R. Pariser, Zoheb B. Kazi, Laurie Muldowney, Pranoot Tanpaiboon, Alexandra Freitas, Jeanine Jarnes Utz, Amy S. Rosenberg, Patricia I. Dickson, Laurence A. Turka, Rekha Abichandani, Donna Griebel, Priya S. Kishnani, Jessica J. Lee, Maureen Dewey, Chester B. Whitley, Derek Gavin
Publikováno v:
Molecular genetics and metabolism, vol 117, iss 2
Kishnani, PS; Dickson, PI; Muldowney, L; Lee, JJ; Rosenberg, A; Abichandani, R; et al.(2016). Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction. MOLECULAR GENETICS AND METABOLISM, 117(2), 66-83. doi: 10.1016/j.ymgme.2015.11.001. UCLA: Retrieved from: http://www.escholarship.org/uc/item/59n944ht
Kishnani, PS; Dickson, PI; Muldowney, L; Lee, JJ; Rosenberg, A; Abichandani, R; et al.(2016). Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction. MOLECULAR GENETICS AND METABOLISM, 117(2), 66-83. doi: 10.1016/j.ymgme.2015.11.001. UCLA: Retrieved from: http://www.escholarship.org/uc/item/59n944ht
The US Food and Drug Administration (FDA) and National Organization for Rare Disease (NORD) convened a public workshop titled "Immune Responses to Enzyme Replacement Therapies: Role of Immune Tolerance Induction" to discuss the impact of anti-drug an
Autor:
Larissa Lapteva, Anne R Pariser
Publikováno v:
Journal of Investigative Medicine. 64:376-382
Background The Food and Drug Administration (FDA)'s Center for Drug Evaluation and Research (CDER) receives about 1500 initial Investigational New Drug applications (INDs) per year. In the first 30 days after initial IND submission, FDA conducts a re
Autor:
Hong H. Vu, Anne R. Pariser
Publikováno v:
Therapeutic Innovation & Regulatory Science. 49:434-442
Prior studies suggested that holding preinvestigational new drug application (PIND) meetings with FDA has a positive effect on clinical development time (CDT).New product marketing applications submitted to FDA CDER during fiscal years 2008-2012 were
Autor:
Anne R. Pariser, William A. Gahl
Publikováno v:
Journal of General Internal Medicine. 29:804-807
Rare diseases play a leading role in innovation and the advancement of medical and pharmaceutical science. Most rare diseases are genetic disorders or atypical manifestations of infectious, immunologic, or oncologic diseases; they all provide opportu
Autor:
Kathryn O’Connell, Anne R. Pariser
Publikováno v:
Expert Opinion on Orphan Drugs. 2:869-875
Background: Limited prevalence diseases present challenges for drug development and for the amount of safety information available to prescribers and patients when a drug is approved for marketing.Objective: To inform the issue of safety populations
Publikováno v:
Expert Opinion on Orphan Drugs. 1:507-510
The United States Food and Drug Administration (FDA) has long recognized the key role it plays in ensuring patients have timely access to safe and effective drugs. Over the past 25 years, FDA has developed multiple programs, including Fast Track, Acc