Background:
The global expansion of gene therapy for spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) has driven cross-border medical travel from countries with limited access or restrictive eligibility criteria. Post-therapy outcomes are influenced by factors beyond the treatment itself-including health system readiness, clinician communication, and family support structures.
Objective:
To describe real-world barriers to post-therapy continuity of care and highlight collaborative strategies implemented to enhance long-term follow-up for internationally treated patients.
Result:
We reviewed clinical and communication data for 173 children (159 SMA, 14 DMD) from 17 countries who received gene therapy at Medcare Hospital, Dubai, between 2020 and 2025. Data were obtained from medical records, parental interviews, teleconsultations, and correspondence with home-country providers to assess barriers to follow-up and determinants of care continuity.
Families frequently travelled because of therapy unavailability or local regulatory constraints, often relying on charitable or crowdfunding support. Major challenges included fragmented follow-up, limited local neuromuscular expertise, and parental hesitancy to disclose prior therapy, fearing loss of access to adjunctive treatments. Outcomes were strongly shaped by parental education, healthcare literacy, and access to a local clinician for ongoing supervision.
Proactive interventions included structured discharge summaries routinely shared with local pediatric neurologists (with parental consent) before families’ return to their respective home countries, ensuring continuity of care. A dedicated gene-therapy coordinator and language translators (e.g. Turkish and Russian) facilitated communication between families and clinical teams, arranged teleconsultations, and supported pre- and post-therapy guidance.
Mapping of regional clinicians was initiated to direct families without established specialists toward appropriate follow-up resources. Direct outreach and visits to countries like Turkey, Kyrgyzstan, and Russia further strengthened collaborative networks.
Conclusion:
Cross-border gene therapy demands global responsibility. Long-term success depends on transparent communication, clinician collaboration, and harmonized care pathways that ensure equitable and continuous outcomes regardless of geography or economic background.