Angelman Syndrome News

• Parenting a child with Angelman syndrome requires constant vigilance August 11, 2026
  There are some things I never expected would become a normal part of parenting. One of the first questions I ask someone who’s been looking after Jude, our 3-year-old son who lives with Angelman syndrome, for an extended period isn’t always, “How was his day?” Sometimes it’s, “Did he do a poo?” Constipation is an […]
• Phase 3 trial begins testing potential Angelman syndrome treatment August 10, 2026
  The Phase 3 BEACON clinical trial, which is testing the experimental therapy rugonersen in people with Angelman syndrome, has begun dosing participants. Oak Hill Bio, the company developing rugonersen and sponsoring the trial, announced that the first participant had been dosed in late July. The trial is currently recruiting participants at sites in California and […]
• Our nonverbal son’s hospitalization taught us some important lessons July 31, 2026
  It’s the middle of winter here in Australia. Runny noses, coughs, and viruses have made their way through households across the country, and ours was no exception. So it wasn’t a surprise when our 3-year-old son, Jude, who lives with Angelman syndrome, recently developed a fever and had a seizure. Fever is a common seizure […]
• Angelman clinical trial enrolls children to test new treatment July 13, 2026
  Enrollment of the first group of participants in a Phase 3 clinical trial testing obudanersen in people with Angelman syndrome is complete, with 136 children taking part in the study to determine whether the treatment can improve communication. The REVEAL study (NCT06914609) will evaluate the therapy in kids and adults. Ionis Pharmaceuticals, the company developing obudanersen and […]
• Angelman syndrome seizures are unpredictable, but we carry on June 9, 2026
  It was the day before a long weekend, and we were supposed to leave that afternoon for a family road trip we’d planned six months earlier. My husband, Tom, decided to take Jude, our 3-year-old son with Angelman syndrome, out that morning to buy a pair of glasses after learning earlier in the week that […]
• $32.5M to advance rugonersen into late-stage testing for Angelman June 8, 2026
  Oak Hill Bio has secured $32.5 million in financing to advance rugonersen, an experimental treatment for Angelman syndrome once in the pipeline at Roche, into late-stage clinical testing. The planned Phase 3 trial was dubbed pivotal by the developer in a company press release, which called rugonersen a “potentially best-in-class” treatment. The rare disease therapeutics […]
• 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 […]