Ultragenyx Pharmaceutical Inc. announced today that the first patient(s) has/have been dosed in the pivotal Phase 3 Aspire study evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide (ASO) for Angelman syndrome. Check clinicaltrials.gov for recruitment status and participation criteria. […]
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Ben Philpot, PhD and his lab at UNC Chapel Hill have identified a small molecule that could be a potential gene therapy treatment for AS. The ASF first funded Dr. Philpot’s pilot study to partner with Pfizer to evaluate over […]
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Exciting news for the Angelman syndrome community! Ionis’ Angelman syndrome program, ION582, has just shared positive preliminary results from the Phase 1/2a HALOS clinical trial. Ionis will be providing an update at the upcoming Angelman Syndrome Foundation meeting in July. […]
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See a letter to the Angelman community. Ultragenyx reported positive interim data from the ongoing Phase 1/2 study in patients living with deletion genotype Angelman syndrome after treatment with investigational GTX-102. From company press release: “The totality of these […]
Read moreAURORA, IL – Angelman Syndrome Foundation (ASF) proudly announces the launch of the Jacob Pritzker Fellowship Program in honor of late board president Fred Pritzker. The Jacob Pritzker Fellowship Program was created to address the ever-increasing need for more physicians who […]
Read moreResearch Investigates Health Issues in Adults with Angelman Syndrome Dr. Ron Thibert, the well-known Angelman syndrome clinician and champion of the low-glycemic index dietary seizure treatment, and Dr. Anna Larson, both of Massachusetts General Hospital, published the findings from their clinical […]
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Angelman Syndrome Foundation (ASF) proudly announces the collaboration with OVID Therapeutics to advance understanding of AS via a contribution of crucial data collected in Ovid’s clinical trial for Angelman syndrome to the LADDER database. “The most important gift that industry […]
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Angelman Syndrome Foundation (ASF) and Dup15q Alliance proudly announce their strategic partnership to launch the LADDER Learning Network, a new medical network specifically designed for individuals with Angelman syndrome (AS) and dup15q—two rare disorders affecting the q-arm of the […]
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