Objectives

After participating in this educational activity, attendees should be able to:

  1. Describe how adeno-associated virus (AAV) vectors are used to deliver gene therapies for pediatric rare diseases.

  2. Explain how natural history studies inform patient selection, outcome measures, and trial design for AAV gene therapy studies.

  3. Identify the major safety, ethical, and operational considerations involved in conducting pediatric AAV clinical trials at TiMT.

Presenter(s): 

Eleonora D'Ambrosio, MD
Assistant Professor of Neurology
Medical Director
Translational Institute for Molecular Therapeutics
UMass Chan Medical School

NameRoleFinancial Relationship DisclosuresDiscussion of
Unlabeled/Unapproved Uses of
Drugs/Devices in Presentation?
Dr. Elenora D'AmbrosioSpeaker

Principal investigator for the REGENXBIO
Principal investigator for the INSMED

No

The design and content of Baystate Continuing Interprofessional Education (CE) activities support quality improvement in healthcare and provide fair and balanced views of therapeutic options. Any relevant financial relationships are mitigated prior to the educational activity.

None of the other planners or faculty for this educational activity have relevant financial relationship(s) to disclose with ineligible companies whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.  

Session date: 
10/06/2026 - 8:00am to 9:00am EDT
Location: 
Baystate Medical Center
United States
See map: Google Maps
Add to calendar: 
  • 1.00 ACPE Contact Hours
  • 1.00 AMA PRA Category 1 Credit™
  • 1.00 ANCC Contact Hours
  • 1.00 APA American Psychological Association
    Baystate Health is approved by the American Psychological Association to sponsor continuing education for psychologists. Baystate Health maintains responsibility for this program and its content.
  • 1.00 IPCE Credit Hour(s)
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