ASA members Meeting Hamburg 2025

Membersmeeting ASA Hamburg

The ASA Members Meeting in Hamburg brought together representatives from across Europe and beyond to reflect on progress and plan ahead. Members confirmed over €270,000 in commitments for the 2026 research grant and reaffirmed ASA’s central focus on supporting basic and translational research. Updates were shared on ongoing projects and upcoming conferences, including the Kyoto […]

ASA-Funded Research Published in Human Cell

We are excited to announce the publication of the second scientific paper from our ASA-funded research project, now available in Human Cell. In this study, led by Simão Rocha and colleagues, the team successfully generated three independent induced pluripotent stem cell (iPSC) lines derived from individuals with paternal uniparental disomy of chromosome 15 (patUPD15)—a rare […]

Community Update Phase 3 Trials – Ultragenyx & Ionis

ANGELMAN SYNDROME PHASE 3 TRIALS — COMMUNITY UPDATE (Webinar recorded: 4 Sep 2025) Video here: https://youtu.be/TenTbLp9pIk WHO HOSTED• FAST• ASA ULTRAGENYX — GTX-102 (antisense oligonucleotide targeting UBE3A-ATS)• Phase 1/2 (open-label; ages 4–17, maternal deletion; data cut Sep 2024)– Signals of improvement vs natural history in cognition (Bayley-4), receptive communication, sleep, gross motor, and behavior (ABC-C).– […]

Scientific call for proposals: ASA Grant 2026 

ASA Grant 2026

The Angelman Syndrome Alliance (ASA) would like to announce our sixth grant call, to fund research for Angelman syndrome, open to teams both in Europe and outside of Europe.  Key dates   Background  The Angelman Syndrome Alliance (ASA) is a non-profit organisation registered in the Netherlands, created by a partnership of organizations from around the world […]

Angelman Think tank

Sponsored by ASA & ASF 15 leading researchers from around the world gathered for one dynamic symposium in Lisbon, February 2025. Focused discussions on research priorities and therapeutic goals. The summit marked a pivotal step in forging a global future for research grants — strengthening collaboration, aligning priorities, and building momentum toward meaningful therapeutic breakthroughs […]

Pioneering Research and Collaborations highlighted at 8th ASA Scientific Conference

asa scientific conference 2024 coventry

The 8th ASA scientific conference on Angelman Syndrome in Coventry (UK) on 1st & 2nd August, 2024 brought together top specialists and researchers from around the world, fostering an environment of collaboration and innovation. Esteemed scientists convened to share their latest findings, discuss cutting-edge research, and explore novel therapeutic approaches. The exchange of ideas was […]