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Search results for "rare diseases"

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Viewing 1 - 100 of 295 results by relevance
Consensus Study Report

Rare Diseases and Orphan Products: Accelerating Research and Development  (2010)

Proceedings

Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary  (2009)

Proceedings

Implementing and Evaluating Genomic Screening Programs in Health Care Systems: Proceedings of a Workshop  (2018)

Proceedings

Refining Processes for the Co-Development of Genome-Based Therapeutics and Companion Diagnostic Tests: Workshop Summary  (2014)

Forum on Drug Discovery, Development, and Translation: 2010 Annual Report  (2011)

Proceedings

Relevance of Health Literacy to Precision Medicine: Proceedings of a Workshop  (2016)

Proceedings

Diffusion and Use of Genomic Innovations in Health and Medicine: Workshop Summary  (2008)

Consensus Study Report

Adverse Effects of Vaccines: Evidence and Causality  (2012)

Proceedings

Drug Repurposing and Repositioning: Workshop Summary  (2014)

Roundtable on Translating Genomic-Based Research for Health: 2012 Annual Report  (2013)

Consensus Study Report

NIH Extramural Center Programs: Criteria for Initiation and Evaluation  (2004)

Roundtable on Translating Genomic-Based Research for Health: 2013 Annual Report  (2014)

Roundtable on Translating Genomic-Based Research for Health: 2011 Annual Report  (2012)

Proceedings

Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop  (2017)

Proceedings

Methodologies for Evaluating and Grading Evidence: Considerations for Public Health Emergency Preparedness and Response: Proceedings of a Workshop—in Brief  (2019)

Forum on Drug Discovery, Development, and Translation: 2011 Annual Report  (2012)

Proceedings

Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop  (2019)

Roundtable on Translating Genomic-Based Research for Health: 2008-2009 Annual Report  (2010)

Proceedings

The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop  (2020)

Proceedings

Emerging Technologies and Innovation in Manufacturing Regenerative Medicine Therapies: Proceedings of a Workshop–in Brief  (2024)

Proceedings

Improving the Efficiency and Effectiveness of Genomic Science Translation: Workshop Summary  (2014)

Roundtable on Translating Genomic-Based Research for Health: 2014 Annual Report  (2015)

Proceedings

Realizing the Potential of Genomics across the Continuum of Precision Health Care: Proceedings of a Workshop  (2023)

Proceedings

Implementing a National Cancer Clinical Trials System for the 21st Century: Second Workshop Summary  (2013)

Proceedings

Conflict of Interest and Medical Innovation: Ensuring Integrity While Facilitating Innovation in Medical Research: Workshop Summary  (2014)

Forum on Drug Discovery, Development, and Translation: 10 Year Review: 2011-2020  (2021)

Consensus Study Report

Making Medicines Affordable: A National Imperative  (2018)

Proceedings

The Economics of Genomic Medicine: Workshop Summary  (2013)

Proceedings

Integrating Large-Scale Genomic Information into Clinical Practice: Workshop Summary  (2012)

Proceedings

Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary  (2012)

Proceedings

Assessing Genomic Sequencing Information for Health Care Decision Making: Workshop Summary  (2014)

Proceedings

Genome-Based Diagnostics: Clarifying Pathways to Clinical Use: Workshop Summary  (2012)

Proceedings

Examining the Impact of Real-World Evidence on Medical Product Development: I. Incentives: Proceedings of a Workshop—in Brief  (2018)

Proceedings

Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series  (2019)

Proceedings

Deriving Drug Discovery Value from Large-Scale Genetic Bioresources: Proceedings of a Workshop  (2016)

Consensus Study Report

Selected Heritable Disorders of Connective Tissue and Disability  (2022)

Consensus Study Report

Living with ALS  (2024)

Proceedings

Assuring Data Quality and Validity in Clinical Trials for Regulatory Decision Making: Workshop Report  (1999)

Forum on Drug Discovery, Development, and Translation: 2015 Annual Report  (2016)

Proceedings

Genome-Based Diagnostics: Demonstrating Clinical Utility in Oncology: Workshop Summary  (2013)

Proceedings

Innovation in Drug Research and Development for Prevalent Chronic Diseases: Proceedings of a Workshop  (2021)

Proceedings

Genomics-Enabled Learning Health Care Systems: Gathering and Using Genomic Information to Improve Patient Care and Research: Workshop Summary  (2015)

Consensus Study Report

Enhancing NIH Research on Autoimmune Disease  (2022)

Proceedings

Challenges and Opportunities in Using Residual Newborn Screening Samples for Translational Research: Workshop Summary  (2010)

Consensus Study Report

Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act  (2012)

Proceedings

Systems for Research and Evaluation for Translating Genome-Based Discoveries for Health: Workshop Summary  (2009)

Proceedings

Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary  (2016)

Proceedings

Neuroscience Trials of the Future: Proceedings of a Workshop  (2016)

Consensus Study Report

Initial National Priorities for Comparative Effectiveness Research  (2009)

Orphans and Incentives: Developing Technology to Address Emerging Infections  (1997)

Consensus Study Report

The Air Force Health Study Assets Research Program  (2015)

Proceedings

Integrating Research and Practice: Health System Leaders Working Toward High-Value Care: Workshop Summary  (2015)

Proceedings

Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary  (2011)

Proceedings

Applying an Implementation Science Approach to Genomic Medicine: Workshop Summary  (2016)

Consensus Study Report

Assessing Genetic Risks: Implications for Health and Social Policy  (1994)

Proceedings

The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary  (2010)

Proceedings

Drug Research and Development for Adults Across the Older Age Span: Proceedings of a Workshop  (2021)

Proceedings

Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary  (2015)

Consensus Study Report

Knowing What Works in Health Care: A Roadmap for the Nation  (2008)

Consensus Study Report

Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine  (2016)

Proceedings

Rational Therapeutics for Infants and Children: Workshop Summary  (2000)

Consensus Study Report

An Evidence Framework for Genetic Testing  (2017)

Proceedings

Extending the Spectrum of Precompetitive Collaboration in Oncology Research: Workshop Summary  (2010)

Consensus Study Report

Extending Medicare Reimbursement in Clinical Trials  (2000)

Consensus Study Report

Countering Bioterrorism: The Role of Science and Technology  (2002)

Proceedings

Advancing Therapeutic Development for Pain and Opioid Use Disorders Through Public-Private Partnerships: Proceedings of a Workshop  (2018)

Proceedings

Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary  (2014)

Proceedings

Amyotrophic Lateral Sclerosis: Accelerating Treatments and Improving Quality of Life: Proceedings of a Workshop–in Brief  (2023)

Proceedings

Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary  (2012)

Consensus Study Report

Small Clinical Trials: Issues and Challenges  (2001)

Proceedings

Venture Philanthropy Strategies to Support Translational Research: Workshop Summary  (2009)

Proceedings

Innovations in Service Delivery in the Age of Genomics: Workshop Summary  (2009)

Proceedings

Multimodal Biomarkers for Central Nervous System Disorders: Development, Validation, and Clinical Integration: Proceedings of a Workshop  (2023)

Consensus Study Report

Future Uses of the Department of Defense Joint Pathology Center Biorepository  (2012)

Proceedings

Implications of Genomics for Public Health: Workshop Summary  (2005)

Proceedings

Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop  (2018)

Proceedings

Developing a 21st Century Neuroscience Workforce: Workshop Summary  (2015)

Proceedings

Inclusion of Pregnant and Lactating Persons in Clinical Trials: Proceedings of a Workshop  (2023)

Proceedings

Mitigating Health Disparities in Brain Disorders Starting with Basic Science: Proceedings of a Workshop–in Brief  (2024)

Proceedings

Exploring Challenges, Progress, and New Models for Engaging the Public in the Clinical Research Enterprise: Clinical Research Roundtable Workshop Summary  (2003)

Consensus Study Report

A National Cancer Clinical Trials System for the 21st Century: Reinvigorating the NCI Cooperative Group Program  (2010)

Proceedings

Applying Systems Thinking to Regenerative Medicine: Proceedings of a Workshop  (2021)

Proceedings

Accelerating the Development of New Drugs and Diagnostics: Maximizing the Impact of the Cures Acceleration Network: Workshop Summary  (2012)

Proceedings

Generating Evidence for Genomic Diagnostic Test Development: Workshop Summary  (2011)

Proceedings

Risk Factors for Suicide: Summary of a Workshop  (2001)

Proceedings

Innovation and Invention in Medical Devices: Workshop Summary  (2001)

Proceedings

Public Engagement and Clinical Trials: New Models and Disruptive Technologies: Workshop Summary  (2012)

Proceedings

Multi-Center Phase III Clinical Trials and NCI Cooperative Groups: Workshop Summary  (2009)

Adverse Reproductive Outcomes in Families of Atomic Veterans: The Feasibility of Epidemiologic Studies  (1995)

Proceedings

Virtual Clinical Trials: Challenges and Opportunities: Proceedings of a Workshop  (2019)

Consensus Study Report

Regulating Medicines in a Globalized World: The Need for Increased Reliance Among Regulators  (2020)

Proceedings

Sharing Clinical Research Data: Workshop Summary  (2013)

Proceedings

Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop  (2017)

Consensus Study Report

Selected Immune Disorders and Disability  (2022)

Consensus Study Report

The U.S. Commitment to Global Health: Recommendations for the Public and Private Sectors  (2009)

Forum on Drug Discovery, Development, and Translation: 2021 Annual Review  (2022)

Forum on Drug Discovery, Development, and Translation: 2018 Annual Report  (2019)