Demystify Your Disease’s R&D Readiness
Resources
Resources
The Ten Things You Need to Pursue Research For Your Disease: What Are the Options for Research and Why is it so Hard?
Global Genes (link)
How Biomarkers Can Improve the Drug Development Process
U.S. Food and Drug Administration (FDA) (link)
Rare Diseases Registry Program (RaDaR)
National Center for Advancing Translational Sciences (NCATS) (link)
What is Gene Therapy?
U.S. Food and Drug Administration (FDA) (link)
Human Gene Therapy for Rare Diseases
U.S. Food and Drug Administration (FDA) (link)
Global Genes LIVE!: Gene to Therapy: How Individualized Therapies Are Becoming Reality
Global Genes (link)
A Guide to Gene Therapy
Global Genes (link)
Gene Therapy Yesterday, Today and Tomorrow
National Organization for Rare Disorders (NORD) (link)
The Science Behind Gene Therapy
National Organization for Rare Disorders (NORD) (link)
The FDA's Role in Gene Therapy
National Organization for Rare Disorders (NORD) (link)
Understanding the Gene Therapy Process and Aftercare
National Organization for Rare Disorders (NORD) (link)
Life After Gene Therapy
National Organization for Rare Disorders (NORD) (link)
What is genome editing?
National Human Genome Research Institute (NHGRI) (link)
What are genome editing and CRISPR-Cas9?
MedlinePlus (link)
What is CRISPR-Cas9?
Wellcome Genome Campus YourGenome (link)
Somatic Cell Genome Editing
National Center for Advancing Translational Sciences (NCATS) (link)
CAR T Cells: Engineering Patients’ Immune Cells to Treat Their Cancers
National Cancer Institute (NCI) (link)
Creating Alerts: PubMed
NIH Library (link)
ClinicalTrials.gov
National Library of Medicine (NLM) (link)
CenterWatch Clinical Trial Listings
CenterWatch (link)
Institutional Review Boards Frequently Asked Questions
U.S. Food and Drug Administration (FDA) (link)
Research Portfolio Online Reporting Tool (RePORTER)
National Institutes of Health (NIH) (link)
List of NIH Institutes, Centers, and Offices
National Institutes of Health (NIH) (link)
Free NIH Email Updates
National Institutes of Health (NIH) (link)
News & Events for Human Drugs
U.S. Food and Drug Administration (FDA) (link)
What's New Related to Drugs (CDER New) and other drug-related topics
U.S. Food and Drug Administration (FDA) (link)
Preclinical Innovation
National Center of Advancing Translational Sciences (NCATS) (link)
Core Technologies
National Center of Advancing Translational Sciences (NCATS) (link)
Monthly NCATS e-newsletter
National Center of Advancing Translational Sciences (NCATS) (link)
Receive NCATS Announcements
National Center of Advancing Translational Sciences (NCATS) (link)
Accomplishments in Genomic Medicine
National Human Genome Research Institute (NHGRI) Genomic Medicine Working Group (GMWG) (link)
2019 Genomic Medicine Year in Review
National Human Genome Research Institute (NHGRI) Genomic Medicine Working Group (GMWG) (link)
News and Events from NCI-funded Research and Programs
National Cancer Institute (NCI) (link)
Genomics and Precision Health Weekly Update
Centers for Disease Control and Prevention (CDC) (link)
PhRMA: Scientific Discoveries
Pharmaceutical Research and Manufacturers of America (PhRMA) (link)
PhRMA: Research and Development
Pharmaceutical Research and Manufacturers of America (PhRMA) (link)
Global Genes Latest News
Global Genes (link)
Global Genes Rare Daily
Global Genes (link)
Global Genes Event Hub
Global Genes (link)
Sign Up for NORD Email Updates
National Organization for Rare Disorders (NORD) (link)
Rare Disease Scientific Workshop
EveryLife Foundation for Rare Diseases (link)