Good Friday Appeal - Royal Children's Hospital, Melbourne
Read More
International News
Angelman Syndrome News – The Web's Daily Resource for Angelman Syndrome News
- MVX-220 for Angelman syndrome earns FDA fast track status October 1, 2025The U.S. Food and Drug Administration (FDA) has granted fast track designation to MVX-220, Mavrix Bio’s investigational gene therapy for Angelman syndrome that is headed into clinical trials. Fast track […] The post MVX-220 for Angelman syndrome earns FDA fast track status appeared first on Angelman Syndrome News.
- Starting seizure medicine has us grappling with anxiety and doubt September 9, 2025Two months ago, we took a step I’d been apprehensive of since Jude, my now 2.5-year-old son, was diagnosed with Angelman syndrome at 16 months, in April 2024. We started […] The post Starting seizure medicine has us grappling with anxiety and doubt appeared first on Angelman Syndrome News.
- Genetic medicine could target Angelman’s root cause September 3, 2025The genetic medicine rugonersen (RO7248824) may be able to target the underlying cause of Angelman syndrome. A study found that the treatment — tested in early clinical trials and expected […] The post Genetic medicine could target Angelman’s root cause appeared first on Angelman Syndrome News.
- ‘Urgent need’ for treatment drives GTX-102 study enrollment August 6, 2025The Phase 3 clinical trial that’s testing an experimental therapy for Angelman syndrome has finished enrolling patients. The Aspire study (NCT06617429), whose results are expected in late 2026, achieved full […] The post ‘Urgent need’ for treatment drives GTX-102 study enrollment appeared first on Angelman Syndrome News.
- Being ‘othered’ is an invisible burden we unfortunately must face July 22, 2025Most days, I’m met with kindness and warmth when I’m out with my 2-year-old son, Jude, who lives with Angelman syndrome. Neighbors see us helping Jude with his gait trainer […] The post Being ‘othered’ is an invisible burden we unfortunately must face appeared first on Angelman Syndrome News.
- Gene therapy GTX-102 granted FDA breakthrough designation July 2, 2025The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to GTX-102 (apazunersen), a gene therapy being developed by Ultragenyx Pharmaceutical for treating Angelman syndrome. “FDA breakthrough therapy […] The post Gene therapy GTX-102 granted FDA breakthrough designation appeared first on Angelman Syndrome News.
- Supporting friends and family while caregiving June 10, 2025Being a caregiver for someone living with Angelman syndrome demands constant physical, mental, and emotional presence. Recently, we’ve become increasingly vigilant and attentive to the needs of our son, Jude, […] The post Supporting friends and family while caregiving appeared first on Angelman Syndrome News.
- First-in-human study of Angelman gene therapy set for this year June 4, 2025The U.S. Food and Drug Administration (FDA) has given Mavrix Bio the go-ahead to start a first-in-human clinical trial testing MVX-220, the company’s experimental gene therapy for Angelman syndrome. The […] The post First-in-human study of Angelman gene therapy set for this year appeared first on Angelman Syndrome News.
- Dietary supplement aids behavior in Angelman syndrome mice: Study May 7, 2025A diet supplemented with octanoic acid safely improved behavior across multiple tests in a mouse model of Angelman syndrome, a study reports. A fatty acid found naturally in breast milk, […] The post Dietary supplement aids behavior in Angelman syndrome mice: Study appeared first on Angelman Syndrome News.
- Advice for parents new to an Angelman syndrome diagnosis April 8, 2025It’s been a year since our son, Jude, was diagnosed with Angelman syndrome (deletion of the UBE3A gene) at 16 months old. The past year has been a roller coaster of appointments, endless […] The post Advice for parents new to an Angelman syndrome diagnosis appeared first on Angelman Syndrome News.