Stories, comments, insights

ASF Blog

February 20, 2024

Rare Disease Week 2024

 FAST and ASF will be joining numerous other rare disease groups on Capitol Hill in person.  While we are in DC,  we invite the Angelman syndrome community to register for  informative sessions with the NIH, Everylife Foundation and FDA and […]

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November 30, 2023

Amanda’s Story of Status

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 […]

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November 3, 2023

Angelman syndrome and the Gut

In 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, […]

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October 27, 2023

Caregiver Awareness Month

November 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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October 16, 2023

Ultragenyx Pharmaceutical Development Pipeline

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 […]

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October 12, 2023

Recruitment Open for the Aldebaran Study of Alogabat

Recruitment 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 […]

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September 22, 2023

Community Update from Ultragenyx

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 […]

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