Skip to main content
Leaderboard Ad
  • National Hemophilia Foundation
  • Steps for Living
  • Victory for Women
  • Better You Know
  • Unite for Bleeding Disorders
  • Unite Your Way
Home
  • Contact Us
  • Subscribe
  • News
  • Events
  • Give
    • Give Now
      • Make a Donation
      • Give Monthly
      • Honor a Loved One
      • Support Research
      • Why NHF?
    • Other Ways to Give
      • Make a Planned Gift
      • Create a Will
      • Give Stock
      • Donor Advised Funds
      • Be a Corporate Partner
      • Donate Your Car
    • Fundraise for NHF
      • Create Your Own Fundraiser
      • Fundraise on Facebook
    • Join Us
      • Red Tie Soiree
      • Red Tie Campaign
      • Find a Walk
      • For Young Professionals

Main navigation

  • Who We Are
  • Bleeding Disorders A-Z
  • Educational Programs
  • Advocacy
  • Research
  • Healthcare Professionals
  • Community Resources
  • Our Story
  • Mission & History
  • What We Do
  • Accomplishments
  • Financial Statements
  • Our Team
  • Employees
  • Board of Directors
  • MASAC
  • Meet the NYLI
  • Nursing Working Group
  • Physical Therapy Working Group
  • Social Work Working Group
  • Our Partners
  • The ACT Initiative
  • Red Tie Society
  • CDC Coop Agreement
  • Careers
  • What Do We Value?
  • Job Openings
  • Volunteer Opportunities
  • Overview
  • Fast Facts
  • What is a Bleeding Disorder?
  • History
  • Types of Bleeds
  • Inhibitors
  • Women and Bleeding Disorders
  • Types
  • Hemophilia A
  • Hemophilia B
  • Von Willebrand Disease
  • Other Factor Deficiencies
  • Inherited Platelet Disorders
  • Treatment
  • Comprehensive Medical Care
  • Treatment Guidelines (MASAC)
  • Current Treatments
  • Future Therapies
  • Clinical Trials
  • Healthcare Coverage
  • Choosing an Insurance Plan
  • Private Insurance
  • Public Insurance
  • Health Insurance Toolkit
  • Education
  • Inhibitor Education
  • Online Education
  • Rare Bleeding Disorders
  • Von Willebrand Disease
  • Mental Health
  • Gene Therapy Education
  • Women
  • Live Workshops
  • Training
  • Steps for Living Facilitation Training
  • Youth Leadership (NYLI)
  • Youth International Twinning Partnership
  • Outreach
  • Undiagnosed
  • Guías Culturales
  • Health Equity Working Group
  • Federal Priorities
  • Access to Care
  • Federal Programs
  • Medicare
  • Medicaid
  • Blood & Blood Product Safety
  • State Priorities
  • Utilization Management
  • Patient Out-of-Pocket Expenses
  • Medicaid
  • How You Can Take Action
  • Register to Vote
  • Washington Days
  • Advocacy Do’s and Don’ts
  • 6 Steps for Grassroots Advocacy
  • Tell Your Story
  • Research Projects
  • Funded by NHF
  • Presented at Our Conference
  • Fund Your Research
  • Judith Graham Pool Postdoctoral Research Fellowship
  • Career Development Award
  • Bridge Award
  • Innovative Investigator Research Award
  • Jeanne Marie Lusher Diversity Fellowship
  • Nursing Excellence Fellowship
  • Physical Therapy Excellence Fellowship
  • Social Work Excellence Fellowship
  • Community Voices in Research
  • What is CVR?
  • How & Why Should I Participate?
  • Impact on Research
  • Frequently Asked Questions
  • Guidelines on Care
  • Comprehensive Care
  • MASAC Documents
  • Products Licensed (US)
  • Emergency Management
  • Education & Resources
  • BDC: Provider Enduring Web Activity
  • NHF-Takeda Clinical Fellowship Program
  • Online Education Activities for Providers
  • Live & Online Learning (Partners)
  • Rare Coagulation Resource Room
  • Peer-reviewed Journals
  • NHF Publications
  • Other Associations
  • Allied Healthcare
  • Nursing
  • Physical Therapy
  • Social Work
  • Managed Care & Payers
  • Collaborating on Coverage
  • Quality of Care Guidelines
  • CME/CE Webcast Series
  • Educational Web Portal
  • Quality Improvement & Cost Management
  • Request Information
  • Call Our Information Center
  • Read Our Publications
  • Get HemAware Magazine
  • Subscribe for Email Updates
  • Resources Near You
  • NHF Chapters
  • Hemophilia Treatment Centers
  • Clinical Trials
  • Bleeding Disorders Camps
  • Financial Assistance
  • Scholarships
  • Patient Assistance Programs
Home
  • Who We Are
    • Our Story
      • Mission & History
      • What We Do
      • Accomplishments
      • Financial Statements
    • Our Team
      • Employees
      • Board of Directors
      • MASAC
      • Meet the NYLI
      • Nursing Working Group
      • Physical Therapy Working Group
      • Social Work Working Group
    • Our Partners
      • The ACT Initiative
      • Red Tie Society
      • CDC Coop Agreement
    • Careers
      • What Do We Value?
      • Job Openings
      • Volunteer Opportunities
  • Bleeding Disorders A-Z
    • Overview
      • Fast Facts
      • What is a Bleeding Disorder?
      • History
      • Types of Bleeds
      • Inhibitors
      • Women and Bleeding Disorders
    • Types
      • Hemophilia A
      • Hemophilia B
      • Von Willebrand Disease
      • Other Factor Deficiencies
      • Inherited Platelet Disorders
    • Treatment
      • Comprehensive Medical Care
      • Treatment Guidelines (MASAC)
      • Current Treatments
      • Future Therapies
      • Clinical Trials
    • Healthcare Coverage
      • Choosing an Insurance Plan
      • Private Insurance
      • Public Insurance
      • Health Insurance Toolkit
  • Educational Programs
    • Education
      • Inhibitor Education
      • Online Education
      • Rare Bleeding Disorders
      • Von Willebrand Disease
      • Mental Health
      • Gene Therapy Education
      • Women
      • Live Workshops
    • Training
      • Steps for Living Facilitation Training
      • Youth Leadership (NYLI)
      • Youth International Twinning Partnership
    • Outreach
      • Undiagnosed
      • Guías Culturales
      • Health Equity Working Group
  • Advocacy
    • Federal Priorities
      • Access to Care
      • Federal Programs
      • Medicare
      • Medicaid
      • Blood & Blood Product Safety
    • State Priorities
      • Utilization Management
      • Patient Out-of-Pocket Expenses
      • Medicaid
    • How You Can Take Action
      • Register to Vote
      • Washington Days
      • Advocacy Do’s and Don’ts
      • 6 Steps for Grassroots Advocacy
      • Tell Your Story
  • Research
    • Research Projects
      • Funded by NHF
      • Presented at Our Conference
    • Fund Your Research
      • Judith Graham Pool Postdoctoral Research Fellowship
      • Career Development Award
      • Bridge Award
      • Innovative Investigator Research Award
      • Jeanne Marie Lusher Diversity Fellowship
      • Nursing Excellence Fellowship
      • Physical Therapy Excellence Fellowship
      • Social Work Excellence Fellowship
    • Community Voices in Research
      • What is CVR?
      • How & Why Should I Participate?
      • Impact on Research
      • Frequently Asked Questions
  • Healthcare Professionals
    • Guidelines on Care
      • Comprehensive Care
      • MASAC Documents
      • Products Licensed (US)
      • Emergency Management
    • Education & Resources
      • BDC: Provider Enduring Web Activity
      • NHF-Takeda Clinical Fellowship Program
      • Online Education Activities for Providers
      • Live & Online Learning (Partners)
      • Rare Coagulation Resource Room
      • Peer-reviewed Journals
      • NHF Publications
      • Other Associations
    • Allied Healthcare
      • Nursing
      • Physical Therapy
      • Social Work
    • Managed Care & Payers
      • Collaborating on Coverage
      • Quality of Care Guidelines
      • CME/CE Webcast Series
      • Educational Web Portal
      • Quality Improvement & Cost Management
  • Community Resources
    • Request Information
      • Call Our Information Center
      • Read Our Publications
      • Get HemAware Magazine
      • Subscribe for Email Updates
    • Resources Near You
      • NHF Chapters
      • Hemophilia Treatment Centers
      • Clinical Trials
      • Bleeding Disorders Camps
    • Financial Assistance
      • Scholarships
      • Patient Assistance Programs
  • Contact Us
  • Subscribe
  • News
  • Events
  • Give
    • Give Now
      • Make a Donation
      • Give Monthly
      • Honor a Loved One
      • Support Research
      • Why NHF?
    • Other Ways to Give
      • Make a Planned Gift
      • Create a Will
      • Give Stock
      • Donor Advised Funds
      • Be a Corporate Partner
      • Donate Your Car
    • Fundraise for NHF
      • Create Your Own Fundraiser
      • Fundraise on Facebook
    • Join Us
      • Red Tie Soiree
      • Red Tie Campaign
      • Find a Walk
      • For Young Professionals
National Hemophilia Foundation Steps for Living Victory for Women Better You Know Unite for Bleeding Disorders Unite Your Way

Resource Guide

  1. Home
  2. Bleeding Disorders A-Z
  3. Treatment
  4. Future Therapies
  5. Resource Guide

Share

FREQUENTLY ASKED QUESTIONS ON GENE THERAPY
Insights FAQ
GENE THERAPY GLOSSARY OF TERMS
Insights Glossary
Gene Therapy – Questions for Your Provider
Gene Therapy – Questions for Your Provider

Main navigation

  • Frequently Asked Questions
  • Glossary
  • Resource Guide
  • Webinars and Videos
Advertisement

Contact us

Brendan Hayes
Brendan Hayes
Director of Education, Innovative Therapies
mail bhayes@hemophilia.org
phone (469) 404-8467

Stay in the know

Subscribe to our newsletter to keep up to speed about everything bleeding disorders.
Lists:

Contact us

7 Penn Plaza Suite 1204, New York, NY 10001, United States
212.328.3700
212.328.3777

About NHF

  • Our Mission
  • What We Do
  • Our Team
  • Join Us
  • Request Information

Resources

  • NHF Chapters
  • HTC Search
  • Clinical Trials
  • Scholarships
  • Camp Near You

Give

  • Donate
  • Fundraise for NHF
  • Other Ways to Give
  • Volunteer
  • Why NHF?

Legal

  • Privacy Policy
  • Medical Disclaimer
  • Gift Acceptance
  • Conflict of Interest
  • Whistleblower
National Hemophilia Foundation logo

©  National Hemophilia Foundation
All rights reserved. 

Charity Navigator