Preparing and supporting children undergoing gene therapy

Little Journey partnered with a global pharmaceutical company to tailor our support solution for children with Duchenne Muscular Dystrophy (DMD) participating in gene therapy research.

DMD is a rare genetic disorder characterised by progressive muscle degeneration and weakness due to a fault in the dystrophin gene. Dystrophin is a protein that helps keep muscle cells intact.

Gene therapy is a new treatment which aims to introduce a working version of a gene into cells in place of the faulty one. For individuals with DMD, this therapy has the potential to protect the muscles and prevent disease progression. 

Screening & Enrolment

Gene therapy research presents unique challenges in screening and enrolment. Families pin their hope on the potential treatment, but not all will meet the inclusion criteria. For those who are eligible, enrolling means they forfeit any future gene therapy options. This creates a daunting "all or nothing" scenario for families. Our client needed to provide clear information and manage expectations sensitively, supporting ineligible participants while empowering eligible families to make informed enrolment decisions.

Engagement & Retention

Retaining participants during the monitoring phase is vital for successful data collection. However, this period brings uncertainty for families, who may worry about adverse events, effectiveness, and disruption to their child's life. If the burden feels too high for families, their dropout risk increases. Our client needed to offer ongoing support, motivation, and reassurance to keep families engaged throughout.