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                FDA Meetings, Conferences and Workshops

FDA Meetings, Conferences and Workshops

                        FDA Meetings, Conferences, and Workshops: Past Events
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          FDA Meetings, Conferences and Workshops
          RegenMedEd Webinar: The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases - 03/09/2022FDA Meetings, Conferences and Workshops Webcast
    Event Title
              RegenMedEd Webinar: The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases


                  March 9, 2022

  

    
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      Meeting Information
                Event MaterialsDate:
          
  


     
           

March 9, 2022   

 
 
        
                                      Time: 
                                      
11:00 AM
    - 12:00 PM
ET
                
  Organized By: 
      Organizer
          Center for Biologics Evaluation and Research (CBER)

 

  The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) hosted a public webinar on **Wednesday, March 9, 2022, from 11:00 a.m. - 12:00 p.m. ET.**

The webinar, **The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases**, brought together patients, caregivers, advocates, and other important stakeholders to celebrate and recognize the contributions that patients with rare diseases have made to advancements in regenerative medicine. This event followed Rare Disease Day, an international day for awareness of rare diseases.

There are around 7,000 known rare diseases affecting more than 30 million people in the United States. Many rare diseases are life-threatening, and only a fraction of rare diseases have an available treatment. The FDA remains committed to supporting the development of treatments for rare diseases and engaging with the rare disease community.

Infocard sharing various images of people and patients.
**About the Webinar**

The webinar featured panelists to discuss:

Perspectives from patients living with a rare disease and their experiences participating in clinical research
	Ways patients and caregivers can participate in and advance clinical research for rare diseases
	Why and how regenerative medicine may hold promise for patients with rare diseases
	FDA’s efforts and programs to support the development of rare disease treatments, including regenerative medicine like gene and cell therapiesPanelists included:

Debbie Drell, Director of Membership Services, National Organization for Rare Disorders
	Brian O’Mahony, Chief Executive, Irish Haemophilia Society
	Julienne Vaillancourt, Captain, U.S. Public Health Service Commission Corps & Rare Disease Liaison & Policy Advisor, FDA CBERThe webinar was moderated by Dr. Anne Rowzee, Associate Director for Policy, FDA CBER OTAT.  This webinar was the second in an educational webinar series called RegenMedEd: An FDA CBER OTAT Webinar Series on Regenerative Medicine.

What is Regenerative Medicine?

Regenerative medicine therapies, such as cell and gene therapies, hold promise for transforming medicine and creating options for patients living with difficult, rare, and often incurable diseases. The FDA plays a vital role in facilitating the development of regenerative medicine therapies, as well as in ensuring approved products are safe and effective for patients.

About the RegenMedEd Webinar Series

To further engage patients, caregivers, and advocates on the topic of regenerative medicine, OTAT launched an educational webinar series called RegenMedEd: An FDA CBER OTAT Webinar Series on Regenerative Medicine. The RegenMedEd webinar series aims to bring together patients, caregivers, patient advocates, and FDA staff to discuss foundational information about regenerative medicine therapies, such as gene therapy and cell therapy, and explore opportunities for stakeholders to engage with the FDA to help advance drug development.



Recording

Zoom Webinar Recording for The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases







Event Materials

  Title
            File Type/SizeSlides for The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases
              pdf               (4.81 MB)Transcript for The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases
              pdf               (202.59 KB)

                                                                                  Content current as of:
      08/02/2023
         
                   
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