When Science And Humanity Become Inseparable

This case study spotlights Myrtelle, a gene therapy company focused on rare pediatric neurological diseases, and its collaboration with Mural Health to deliver patient-centered clinical research. In a recent trial, Myrtelle presented transformative results to the FDA—not only through data, but by showcasing the children behind the numbers, whose dramatic improvements brought regulators to tears. Yet the path to this moment was fraught with operational barriers that threatened trial continuity.

Myrtelle’s commitment to equitable access led them to partner with Mural Health. Mural’s platform provided real-time reimbursements, personalized travel coordination, and compassionate, around-the-clock support. This enabled families—regardless of socioeconomic status—to participate in trials critical to advancing gene therapy. Myrtelle and Mural Health redefined what patient-centered gene therapy trials can look like with a collaboration that demonstrates logistical support is not ancillary—it is foundational. Ensuring every family feels seen, supported, and respected is essential for participation, data integrity, and ultimately, therapeutic progress for generations to come.