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 […]
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 […]
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