The past month has been a real rollercoaster for our family with many sleepless nights and a mix of emotions from uncertainty to anxiety due to Non-Convulsive Status Epilepticus (NCSE). I want to share our recent experience in hopes that […]
Read moreIn this exclusive Q&A session, we heard from a remarkable individual who wears multiple hats— a mother, a pediatric gastroenterologist, and a physician-scientist. She is on a mission to improve the lives of those dealing with neurodevelopmental differences. Kara Margolis, […]
Read moreNovember is Caregiver Awareness Month. To honor the loving, hard working, remarkable caregivers of the Angelman community, we’ve compiled a list of resources and services geared toward YOU, the caregiver. Even though caring for an individual with AS can be […]
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Ultragenyx Pharmaceutical provided updates on its development pipeline, including investigational GTX-102 for Angelman syndrome (AS) at Analyst Day held Monday, October 16, 2023. For GTX-102 the antisense oligonucleotide for Angelman syndrome, UGX reported data from the extension cohorts in the […]
Read moreRecruitment is open for the Aldebaran study, sponsored by Roche, to assess an investigational drug (called Alogabat) to potentially treat Angelman syndrome. While it does not involve replacing or unsilencing the UBE3A gene in individuals living with AS, it’s a […]
Read moreUltragenyx Pharmaceuticals, (sponsor of the GTX-102, an Antisense oligonucleotide for the potential treatment of Angelman syndrome) has issued a letter providing an update on their program. See the letter below. Ultragenyx Novato, CA 94949 […]
Read moreRoche has responded to our request by answering questions from the community regarding their recent decision to not initiate any new trials of rugonersen (RO7248824) in Angelman syndrome. Download Questions & Answers from Roche Or scroll down to see the […]
Read moreAs we welcome in a New Year, I want to share with you the accomplishments you’ve helped make possible in 2022. It was an exciting year of growth and success as several programs advanced to improve the lives of all […]
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