Duchenne Muscular Dystrophy Trial In China

Navigating clinical trials for rare diseases can be complex, especially in new regions. This case study details the challenges faced during a Duchenne Muscular Dystrophy (DMD) trial in China, where patient families faced significant obstacles due to the remote locations and demanding travel for weekly infusions. The trial sponsor had to overcome broad skepticism about providing patient services in the country, with additional concerns about language barriers, cultural differences, and technology access. By implementing a patient liaison team based in China, establishing local vendor partnerships, and leveraging a reliable online portal, the trial was able to successfully address these logistical challenges. The outcome was improved continuity of care and enhanced patient engagement, demonstrating that with the right support, complex studies can succeed and keep patients and sites connected.