The 4 patients who tested ADA-positive at or prior to week 12 all had high antibody titers, with peak titers ranging from 46,774 to 302,963 between weeks 86 and 118; the patient who tested ADA-positive at week 20 had a moderate antibody titer that peaked at 2958 at week 84 and fluctuated but showed a decreasing trend through the end of the study, and the patient who had tested ADA-positive at week 60 continued to have low-titer ADAs throughout treatment with a peak titer of 292 at the follow-up visit.
← all excerpts
Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies.
1
—
—