Interviews to improve choices about gene therapy for rare diseases
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This study uses one-on-one interviews to learn what families and clinicians need to make decisions about gene therapy for rare diseases. The goal is to create or improve decision tools that make gene therapy information easier to understand and act on.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are one of: a family member of someone who had gene therapy, a family member offered gene therapy but did not receive it, or a clinician who cared for at least 2 patients
- Your child’s condition (or your patient group) is a rare genetic disease targeted for gene therapy
- You can do a private one-on-one interview by video on a phone or computer with working internet
- You can speak English and are able and willing to give verbal consent
- You must sign a permission form so the study team can contact your care team to verify your diagnosis and gene therapy (if received)
- If you had gene therapy: your care team must confirm whether you received it and what type
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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