Shots:
Ultragenyx has completed enrolment in its P-III (Aspire) trial to assess GTX-102 (apazunersen) in Angelman syndrome pts with confirmation of full maternal UBE3A gene deletion; Aurora study in more ages & genotypes to begin in H2'25
Trial will evaluate GTX-102 (intrathecal, 8mg QM for 3mos., then up to 14mg Q4M) vs a sham treatment…
