Angelman Syndrome
6 videos · updated daily
20:20 Novo and Novartis Trial Setbacks, an Alexander Disease Approval
BioSpace covers Novo Nordisk ending two more late-stage trials of its IL-6 inhibitor, Novartis Phase 3 setbacks in cardiovascular disease and muscular dystrophy, Ultragenyx's Phase 3 Angelman miss, and Ionis' approval in Alexander disease.
44:51 Episode 16 – A Parent's Journey in Drug Development
Drug Development Diaries on a parent's journey through drug development for Angelman syndrome, and how patient advocacy shapes rare disease research.
1:20:01 Allyson Berent on Angelman Syndrome and GTX-102
Dr. Allyson Berent, Chief Science Officer of the Foundation for Angelman Syndrome Therapeutics, discusses parent-led drug development and GTX-102, which in July 2026 was in a fully enrolled global Phase 3 study, for GENEration Hope.
1:00:11 RDDS 2026: Angelman and NPC Drug Development
Advocates, academics and industry trace drug development from gene discovery to commercialization via Angelman syndrome with several candidates in clinical trials and Niemann-Pick Type C with two FDA-approved treatments, at Global Genes' RDDS 2026.
8:39 Angelman Phase 3 Miss, HAE and a Rare Lung Cancer Approval
CheckRare reviews Ultragenyx's Phase 3 Aspire study of apazunersen in Angelman syndrome, which missed its primary and key secondary endpoints, alongside competition in hereditary angioedema and an FDA decision on a lung cancer therapy.
1:47:15 Sessions 3.2 & 4 - RISE Together: Data Sharing Across the Rare Disease Ecosystem
A Duke-Margolis panel with the Foundation for Angelman Syndrome Therapeutics, Amicus and BioMarin discusses what rare disease data should be shared, and the tools and infrastructure to support it.