Frequently Asked Questions

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'GeneTx' is a research study aiming to -

• Identify what information around gene therapy is most needed by patients and families with rare genetic diseases like sickle cell disease, hemophilia, etc.
• Build an online educational platform that will help patients/families to understand and decide on gene therapy vs. other treatment option(s).

Click here to learn more about GeneTx. 

 You are invited to participate in our study if you are -

• A young adult (18-35 years of age) or parent/caregiver of a child with a rare genetic disease who has undergone gene therapy OR
• A young adult (or parent/caregiver of a child) who had been offered but has declined gene therapy or was ultimately not eligible for a trial OR
• A healthcare worker who has provided care to two or more patients receiving gene therapy.

If you are eligible and choose to participate in our study, you will -  

• Take part in an online one-on-one video interview using a personal computer or a mobile device. 
• Share personal viewpoints and informational needs around participating in a clinical trial using gene therapy for rare diseases.  

• Your interview should take about 30-60 minutes. 
• You can complete your interview in person as well if you prefer to do so. 

 Your participation in this study may -

• Help us innovate the first-of-its-kind decision support tool for patients and families with rare diseases.
• Contribute to addressing an urgent need to provide patients and families with accurate information on gene therapy.
• Provide valuable information required to fill an important gap in patient-provider communication around gene therapy.

Note:  All study enrollees will receive $100 as a small token of appreciation for their time and contribution to our study. 

Our study team comprises doctors, nurses, and other researchers who study Sickle Cell Disease and other rare pediatric diseases. 

Principal Investigator:  Liza-Marie Johnson, MD