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Angelman syndrome: A disorder that stops people walking and speaking

Angelman syndrome is a rare disease that disrupts the normal development and function of cells, particularly in the nervous ...
Morningstar

MavriX Bio Receives FDA Fast Track Designation for MVX-220 for Treatment of Angelman Syndrome

MIDDLETON, Mass., Sept. 22, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...
Nasdaq

Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome

Company expects to complete Phase 3 Aspire study in the second half of 2026 Aurora study of GTX-102 in additional ages and genotypes on track to initiate in the second half of 2025 “The accelerated ...