Angelman Syndrome News

• GTX-102 data show continued developmental gains in Angelman May 11, 2026
  Long-term data from a clinical study suggest that the experimental treatment GTX-102 (apazunersen) is associated with continued improvements and developmental gains in children with Angelman syndrome, with results from a Phase 3 trial testing the therapy expected later this year, according to an update from developer Ultragenyx Pharmaceutical. “The latest long-term GTX-102 Phase 1/2 data […]
• What standardized assessments miss about my son with Angelman May 5, 2026
  These days, when friends and family ask about my 3-year-old son, Jude, who lives with Angelman syndrome, my answer comes easily. I tell them about his sweet, affectionate nature, like how he leans into us for cuddles and loves giving high-fives. I talk about his interests, like his newfound love of “The Wiggles.” I proudly […]
• Surgery usually corrects misaligned eyes in kids with Angelman, study finds April 13, 2026
  Surgery aimed at correcting strabismus — a condition in which the eyes are misaligned and do not point in the same direction, causing vision problems — is usually successful in children with Angelman syndrome, according to a new study led by scientists in China. The one-year success rate was at least 70% for the more […]
• The joys and challenges of disability equipment for children with Angelman April 7, 2026
  Over time, our house has slowly filled with medical equipment for our 3-year-old son, Jude, who lives with Angelman syndrome. Some of it was readily available from local shops, such as soft-play equipment to practice climbing. Some arrived after protracted funding applications following assessments and equipment trials, causing frustration when the system moved slowly. Sometimes […]
• Goals that matter most for our child living with Angelman syndrome March 10, 2026
  Like many people, I usually set a few goals at the start of each year, mostly focused on self-care: exercising regularly, getting outdoors, and taking time off when I need a break. But this year, my thinking has shifted. Instead of focusing on what I wanted to change about myself, I thought more intentionally about […]
• Home videos may help doctors evaluate Angelman patient skills March 9, 2026
  Researchers have developed a video-based assessment that can be done at home to evaluate the functional skills of people with Angelman syndrome, saying it may complement clinic-based tests to help doctors better understand what patients are capable of when they aren’t feeling anxious or stressed. The team described the new assessment in the paper, “Development […]
• After hesitation, we finally took steps to address our son’s disrupted sleep February 10, 2026
  When we received our son Jude’s Angelman syndrome diagnosis in April 2024, at just 16 months old, his geneticist warned us that disrupted sleep was to be expected. For many families, sleep disruption is a daily reality of the syndrome. It’s not only exhausting, but also brings the added anxiety of increased seizure risk. Poor […]
• International Angelman Day: Why Feb. 15 matters for families February 9, 2026
  Feb. 15 is International Angelman Day (IAD), an annual event that brings together advocates worldwide to raise awareness and support people affected by Angelman syndrome. IAD was started in 2013 by Angelman parents who saw a need for more connection and support for the rare disease, which is caused by mutations in a gene located […]
• Traveling with a child with Angelman syndrome is easier in the early years January 6, 2026
  After my son, Jude, was diagnosed with Angelman syndrome at 16 months old, I kept hearing the same comment from other seasoned Angelman parents: “Traveling is so much easier when they’re younger.” At the time, I was skeptical. But now that Jude is 3 and has experienced short- and long-haul flights and road trips, I’m […]
• 6 months of keto diet shows promising trends for Angelman children January 5, 2026
  Following a low-sugar, high-fat eating plan — known as a ketogenic, or keto, diet — may benefit children with Angelman syndrome, though more research is needed to know for sure, according to the findings of a new study from Spain. In the small trial, dubbed DIANE, children with Angelman were assigned to follow a ketogenic […]