Angelman Syndrome News – The Web's Daily Resource for Angelman Syndrome News

• Recognizing the importance of exercise for Angelman caregivers November 11, 2025
  After my son, Jude, was diagnosed with Angelman syndrome in April 2024 at 16 months, taking time to focus on my fitness felt like a luxury. Intense emotional processing took over as I cycled through fear, hope, guilt, and exhaustion. Every week was filled with new information, new appointments, and new emotions to manage. Finding […]
• When the world forgets our children November 10, 2025
  This article was provided by our partner, the Angelman Syndrome Foundation. It has been reviewed by Bionews for accuracy and relevance. The views and opinions expressed are those of the author and do not necessarily reflect the views of Bionews or Angelman Syndrome News. I’m writing this because I don’t know where else to put […]
• Dosing begins in broader trial of Angelman syndrome therapy November 3, 2025
  The first person has been dosed in a Phase 3 clinical trial testing GTX-102 (apazunersen), Ultragenyx Pharmaceutical’s experimental therapy, in a broad population of Angelman syndrome patients. The study, dubbed Aurora (NCT07157254), is the second Phase 3 trial of GTX-102 in Angelman patients. The first, called Aspire (NCT06617429), completed the enrollment of 129 children and teenagers with […]
• The guessing game involved in caring for a nonverbal child October 7, 2025
  My 2-year-old son, Jude, lives with Angelman syndrome and is nonverbal, so I’ve had to become more attuned to the subtle ways he communicates his thoughts and feelings. Sometimes he clearly expresses how he’s feeling, such as by laughing while playing peekaboo, spinning in a sensory swing, or (gasp) listening to farts. Other times, I’ll […]
• MVX-220 for Angelman syndrome earns FDA fast track status October 1, 2025
  The 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 designation aims to facilitate the clinical development and regulatory review of therapies intended to treat serious conditions with an unmet medical need. The status affords […]
• Starting seizure medicine has us grappling with anxiety and doubt September 9, 2025
  Two 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 seizure medicine. We knew seizures were common in people with Angelman syndrome, but the experience still felt surreal when they arrived. Although we educated ourselves […]
• Genetic medicine could target Angelman’s root cause September 3, 2025
  The 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 to enter Phase 3 trials early next year — restored UBE3A protein activity in mouse and primate models of the syndrome. The study, “Angelman syndrome […]
• ‘Urgent need’ for treatment drives GTX-102 study enrollment August 6, 2025
  The 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 enrollment, with about 129 participants, barely half a year after it began enrolling last December, according to Ultragenyx Pharmaceutical, which is developing GTX-102 (apazunersen) and […]
• Being ‘othered’ is an invisible burden we unfortunately must face July 22, 2025
  Most 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 and kneel down to his level to smile and encourage him. Friends and family show genuine interest in learning about Jude’s augmentative and alternative communication […]
• Gene therapy GTX-102 granted FDA breakthrough designation July 2, 2025
  The 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 designation underscores both the urgent need for an effective treatment for patients and families affected by Angelman syndrome and the clinically meaningful results demonstrated to […]