AnneRugglesPariserMD

PubMed

• 2 citations
  Recent Advances in Systems and Network Medicine: Meeting Report from the First International Conference in Systems and Network Medicine.
  Emma Kurnat-Thoma, Ancha Baranova, Pat Baird, Elia Brodsky, Atul J. Butte, Amrita K. Cheema, Feixiong Cheng, Shuchismita Dutta, Christina Grant, James Giordano, Anke H...> ;Systems Medicine. 2020 Feb 26
• 31 citations
  Understanding the evolving phenotype of vascular complications in telomere biology disorders
  Cecilia P. Higgs, Yanick J. Crow, Denise M. Adams, Emmanuel Chang, Don Hayes, Utz Herbig, James N. Huang, Ryan Himes, Kunal Jajoo, F. Brad Johnson, Susan D. Reynolds, ...> ;Angiogenesis. 2019 Feb 1
• 65 citations
  From scientific discovery to treatments for rare diseases - the view from the National Center for Advancing Translational Sciences - Office of Rare Diseases Research.
  Petra Kaufmann, Anne Pariser, Christopher P. Austin> ;Orphanet Journal of Rare Diseases. 2018 Nov 6
• 27 citations
  Representation of Women and Minorities in Clinical Trials for New Molecular Entities and Original Therapeutic Biologics Approved by FDA CDER from 2013 to 2015.
  Alice Chen, Hilary Wright, Hawi Itana, Merina Elahi, Guoxing Soon, Anne R. Pariser, Emmanuel O. Fadiran, Ayomide Igun> ;Journal of Women's Health. 2017 Oct 19
• 53 citations
  Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction
  Priya S. Kishnani, Patricia I. Dickson, Laurie Muldowney, Jessica J. Lee, Amy S. Rosenberg, Rekha Abichandani, Jeffrey A. Bluestone, Barbara K. Burton, Maureen Dewey, ...> ;Molecular Genetics and Metabolism. 2016 Feb 1
• 5 citations
  Investigational New Drug applications: a 1-year pilot study on rates and reasons for clinical hold
  Larissa Lapteva, Anne R Pariser> ;Journal of Investigative Medicine. 2016 Feb 1
• 1 citations
  Pre–Investigational New Drug Meetings With the FDA: Evaluation of Meeting Content and Characteristics of Applications for New Drug and Biologic Products
  Hong H. Vu, Anne R. Pariser> ;Therapeutic Innovation & Regulatory Science. 2015 May 1
• 25 citations
  Important Role of Translational Science in Rare Disease Innovation, Discovery, and Drug Development
  Anne R. Pariser, William A. Gahl> ;Journal of General Internal Medicine. 2014 Jul 17
• 150 citations
  Phenylketonuria Scientific Review Conference: state of the science and future research needs.
  Kathryn M. Camp, Melissa A. Parisi, Phyllis B. Acosta, Gerard T. Berry, Deborah A. Bilder, Nenad Blau, Olaf Bodamer, Jeffrey P. Brosco, Christine Brown, Alberto Burlin...> ;Molecular Genetics and Metabolism. 2014 Jun 1
• 30 citations
  Cross-sector sponsorship of research in eosinophilic esophagitis: a collaborative model for rational drug development in rare diseases.
  Robert P. Fiorentino, Gumei Liu, Anne R. Pariser, Andrew E. Mulberg> ;The Journal of Allergy and Clinical Immunology. 2012 Sep 1
• 11 citations
  Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010.
  Anne R. Pariser, Daniel J. Slack, Larry Bauer, Catherine A. Warner, LaRee Tracy> ;Drug Discovery Today. 2012 Aug 1
• 19 citations
  The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease.
  Emanuela Lacana, Lynne Yao, Anne R. Pariser, Amy S. Rosenberg> ;American Journal of Medical Genetics. Part C, Seminars in Medical Genetics. 2012 Feb 15
• 22 citations
  Clinical pharmacology as a cornerstone of orphan drug development.
  Edward D. Bashaw, Shiew-Mei Huang, Timothy R. Coté, Anne R. Pariser, Christine Garnett, Gilbert J. Burckart, Lei Zhang, Angela Y. Men, Christine D. Le, Rosane Charlab,...> ;Nature Reviews. Drug Discovery. 2011 Oct 31
• 11 citations
  Regulatory considerations for developing drugs for rare diseases: orphan designations and early phase clinical trials.
  Anne R. Pariser, Kui Xu, John Milto, Timothy R. Coté> ;Discovery Medicine. 2011 Apr 1
• 18 citations
  Research challenges in central nervous system manifestations of inborn errors of metabolism
  Patricia I. Dickson, Anne R. Pariser, Stephen C. Groft, R. W. Ishihara, D. E. McNeil, Danilo A. Tagle, Donna Griebel, Stephen G. Kaler, Jonathan W. Mink, Elsa Shapiro,...> ;Molecular Genetics and Metabolism. 2011 Mar 1
• 14 citations
  Training the Translational Scientist
  Rebecca D. Jackson, Sherine Gabriel, Anne Pariser, Peter Feig> ;Science Translational Medicine. 2010 Dec 22
• 21 citations
  Accelerating orphan drug development.
  Timothy R. Coté, Kui Xu, Anne R. Pariser> ;Nature Reviews. Drug Discovery. 2010 Dec 1
• 13 citations
  Therapies for inborn errors of metabolism: what has the orphan drug act delivered?
  Sonali S Talele, Kui Xu, Anne R. Pariser, M. Miles Braun, Sheiren Farag-El-Massah, M. Ian Phillips, Barry H. Thompson, Timothy R. Coté> ;Pediatrics. 2010 Jul 1
• 117 citations
  Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment
  Jinhai Wang, Jay Lozier, Gibbes R. Johnson, Susan L. Kirshner, Daniela Verthelyi, Anne R. Pariser, Elizabeth Shores, Amy S. Rosenberg> ;Nature Biotechnology. 2008 Aug 1
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