Clinical Trial: Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
Study Status: Completed
Recruit Status: RECRUITING
Condition: Sickle Cell Disease
Study Type: OBSERVATIONAL
Official Title: Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
Brief Summary:
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques.Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.
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