Ultragenyx 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 […]
Read moreAs we come closer to Giving Tuesday, ASF is excited to announce we will yet again have a donation match. Through the generosity of the Harvey family, donations will be matched up to $100,000. That means we can raise $200,000 […]
Read moreEver since being diagnosed with Angelman syndrome, we longed to be able to take our children to an Angelman Syndrome Clinic here in the USA. We decided on the clinic at Children’s Hospital Colorado with Dr. Jessica Duis and […]
Read moreOur daughter, Maddie Sargent, was diagnosed with Angelman syndrome in January 2020. Mystified, terrified and on the brink of a worldwide pandemic, we were determined to find the most experienced providers to care for our girl. We took Maddie to […]
Read moreI am so grateful to be able to share our story with you. It starts out probably very similar to yours. It was Nathan’s seizures that led us to his Angelman syndrome diagnosis. Over 80% of individuals with AS develop epilepsy. […]
Read moreWhat a year it has been for our community! As I reflect back on 2021, I am filled with mixed emotions. On one hand, it has been such an amazing year for the ASF and the community when it comes […]
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