About this study
GENETX will help us understand the information that patients and families need about gene therapy for rare genetic diseases, in order 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 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:
- 18–35 years old with a rare genetic disease who had gene therapy
- Parent or caregiver of a child with a rare genetic disease who had gene therapy
- 18–35 years old, who was offered gene therapy, but did not have it because they were not eligible or did not want it
- Parent or caregiver of a child who was offered gene therapy, but did not have it because they were not eligible or did not want it
- Health care worker who cared for 2 or more patients who had gene therapy