Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Shots: The US FDA has granted BTD to ION582 (UBE3A protein expression stimulant) for the treatment of Angelman syndrome Designation was based on P-I/II (HALOS) trial assessing ION582 in pts with Angelman syndrome, which showed improvements across all functional domains incl. communication, cognition & motor function; safety was favorable Additionally, ION582 is being investigated in…

ByRidhi RastogiSeptember 12, 2025

Ultragenyx Completes Patient Enrolment in P-III (Aspire) Trial of GTX-102 for Angelman Syndrome

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…

ByRidhi RastogiAugust 1, 2025

MavriX Bio Reports the US FDA’s IND Clearance of MVX-220 for Angelman Syndrome

Shots: The US FDA has approved IND application of MVX-220 for the treatment of Angelman Syndrome (AS) to initiate P-I/II (ASCEND-AS) trial, with first pts dosing expected in H2’25 The P-I/II trial will evaluate the safety, tolerability, & effectiveness of MVX-220 in both adult & pediatric pts with multiple AS genotypes, incl. rare types like uniparental…

ByRidhi RastogiMay 13, 2025

Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Ultragenyx Completes Patient Enrolment in P-III (Aspire) Trial of GTX-102 for Angelman Syndrome

MavriX Bio Reports the US FDA’s IND Clearance of MVX-220 for Angelman Syndrome

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