Gospel Enyindah-Asonye's questions to Regenxbio Inc (RGNX) leadership • Q3 2024
Question
Gospel Enyindah-Asonye, on behalf of Vikram Purohit at Morgan Stanley, asked for guidance on how best to interpret the upcoming functional data for the RGX-202 Duchenne program.
Answer
Dr. Steve Pakola, Chief Medical Officer, explained that the company will assess the totality of the data, including standard timed function tests like the 10-meter walk/run, NSAA scores, and caregiver-reported outcomes. CEO Curran Simpson added that their analysis will be supported by comparisons to external controls and substantial natural history databases.