As with other clinical trials examining orphan or ultra-rare indications, it's often a challenge to identify, screen, and enroll eligible patients. And while there is significant interest from parents, caregivers, and physicians to provide a precision medicine to this important unmet medical need, this is also a young and fragile patient population, which sometimes makes travel to clinical sites difficult.

Nov 8, 2022 / receipt, needs a key / Dr. Chris Kenney, Chief Medical Officer | earnings call 2022-11-08 | transcript fd004528-d27f-4b8f-b51d-2153f329471b | chars 5697-6106 | segment 5