Meet Maddie, the Children’s Mercy patient battling Angelman syndrome who exudes positivity

Angelman syndrome is a rare genetic disorder that impacts brain development, speech, movement, and learning.
ABC12

Montrose family raising money for 6-year-old boy with Angelman Syndrome

MONTROSE, Mich. (WJRT) - A Mid-Michigan family is raising money to help their 6-year-old boy with a rare genetic disorder. After spending a few months looking for action figures and fun items, Kristin ...
Cleveland.com

Local Westlake family helps raise funds for Angelman Syndrome research

The Pinball Lounge at 78th Street Sudios offers an ideal setting for this unique fundraiser. The event runs from 4-6 pm, providing families with an accessible Sunday afternoon activity. True to its ...
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 Pharmaceutical Inc. Doses First Patient in Pivotal Phase 3 Aspire Study for GTX-102 in Angelman Syndrome

Ultragenyx Pharmaceutical Inc. announced the dosing of the first patient in its Phase 3 Aspire study, which is investigating the safety and efficacy of GTX-102, an investigational antisense ...
Seeking Alpha

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 ...