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Core Concepts
The Beginning
Advocating for Your Child
Understanding Interventions
Coping, Family and Relationships
End of Life and Bereavement
Explore
Not sure where to start?
Explore CPN Core Concepts
Learn More
Video Library
Audio Library
Guides
Pathways
Blog
Featured Family
Units
Diagnoses
Events
About
Not sure where to start?
Explore CPN Core Concepts
Learn More
Our Origin
Donors
Annual Report
Parent Champions
Speaking Engagements
Staff and Board
Mobile App
Contribute a Blog
Collaborations and Co-Authorship in Research
Medical Advisory Board
The Ilene Beal and Margaret S Lindsay Courageous Provider Awards (2024 Announcement)
Talk to CPN
En Español
Clinicians/Educators
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MEGAN DONNELL
Megan
Megan, Executive Director Sanfilippo Children’s Foundation. Mother to Isla (10) and Jude (8), Sanfilippo Syndrome
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Clinical Trials: Whatever decision you make, it is going to be right for you.
Clinical Trials: Once treated the road ahead is unknown.
Clinical Trials: As she aged my expectations about what treatment would mean adjusted.
Clinical Trials: Two children screened, one qualifies – antibodies exclude the other.
Clinical Trials: Which child? The decision was made for me.
Clinical Trials: The pioneers and those who come after.
Clinical Trials: Is this really the right thing for my child?
Clinical Trials: What diseases can actually say there is a cure?
Clinical Trials: Waiting for an emerging therapy
Clinical Trials: Prioritizing therapies to meet the eligibility criteria
Clinical Trials: You give up some control when your child is a data point.
Clinical Trials: Restrictions in the Informed Consent and participation can be isolating
Clinical Trials: The logistics and limitations of gene therapy
Clinical Trials: Survivor’s guilt and my place in the community.
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Megan Video Transcripts
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