About this study
GENETX will help us understand the information that patients and families need about gene therapy for rare genetic diseases, to make important decisions about treatment. We want to share correct, easy-to-understand information about gene therapy, so patients and families can speak with their care teams about it.
First, we need to find out what kind of information will help. At the beginning of this study, we will speak with
- Patients with inherited genetic disorders where gene therapy is a possible treatment
- Blood Disorders like Sickle Cell Disease and Hemophilia
- Neuromuscular Diseases like Duchenne Muscular Dystrophy
- Families of these patients
- Health care workers who care for these patients.
By taking part in this study, they will help us create online resources for others.
If you enroll in this study, you will:
- Do an online video interview
- Share your thoughts about what information you need
- Share your concerns about taking part in a gene therapy clinical trial
Eligibility overview
To take part in this study, you must be one of the following:
- Parent or caregiver whose child had gene therapy OR a parent or caregiver of a child who died after receiving gene therapy at least 6 to 24 months before enrollment OR patients age 8 and above who had gene therapy.
- Parent or caregiver of children (patients 8 and above) with a rare genetic disease who were offered a clinical trial but were not eligible or decided against receiving gene therapy.
- Health care worker who has cared for 2 or more patients who had gene therapy