FAST Conference Draws 20 Pharmaceutical Companies and Raises Over $2.2 Million for Research

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Downers Grove, IL – December 7, 2020 – The Foundation for Angelman Syndrome Therapeutics (FAST) today announced that Virtually Unstoppable, their annual Summit & Gala benefitting Angelman syndrome (AS) raised over $2.2 million during their virtual two-day conference. The conference included two science conferences, an educational and best practices workshop, and a mini-Gala featuring award […]

Frequently asked questions regarding the GeneTx KIK-AS Clinical Trial

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GeneTx Biotherapeutics has announced it has received IRB approval from Rush University Medical Center in Chicago, IL to begin the “KIK-AS” (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) Phase 1/2 clinical study of GTX-102. What does this mean? The United States Food and Drug Administration’s Investigational New Drug (IND) program is the means by […]

A message to the Angelman community from GeneTx Biotherapeutics

Two press releases were issued recently with one announcing an Exclusive Option Agreement between GeneTx Biotherapeutics, LLC (“GeneTx”) and  Ultragenyx Pharmaceutical, Inc. (“Ultragenyx”), and the other announcing GeneTx’s lead candidate receiving Orphan Drug Designation (ODD) and Rare Pediatric Drug Designation (RPD). GeneTx would like to take this opportunity to provide additional information to our community about this […]

Angelman Biomarkers and Outcome Measures Alliance and Roche begin patient-centered qualitative research to inform potential outcome measures for Angelman syndrome clinical trials

F. Hoffmann-La Roche Ltd, (Roche), one of the world’s largest pharmaceutical and diagnostics companies, announced today that it has funded the first phase of a study to better understand which symptoms of Angelman syndrome impact families most. The study aims to better understand the impact of Angelman syndrome on patients and their families through interviews […]

Ganaxalone As Potential Therapeutic For Angelman Syndrome

[wpv-autop]Effects of the synthetic neurosteroid ganaxolone on seizure activity and behavioral deficits in an Angelman syndrome mouse model.[/wpv-autop][wpv-autop] By Allyson Berent, DVM, DACVIM Recently published work from Dr. Edwin Weeber’s lab, funded by the FAST (Foundation for Angelman Syndrome Therapeutics), “demonstrates that ganaxolone ameliorates many of the behavioral abnormalities in the adult AS mouse, and […]

FAST Summit Live stream

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Watch all the news and updates from the Science Summit 9-5pm Friday, December 2 (CST) and
the Education Summit on Saturday December 3, 9-4PM.

Maggie Linton Show

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Dr Allyson Berent, the Chief Science Officer at the Foundation for Angelman Syndrome Therapeutics, talks with popular radio journalist Maggie Linton about Angelman Syndrome awareness and the future therapeutics and clinical trials on Sirius XM Urban View.

Ovid Therapeutics Launches First Sponsored Clinical Trial For Angelman Syndrome

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Ovid Therapeutics announces the first ever sponsored clinical trial for Angelman Syndrome. You first heard about Gabaxadol from Dr. Matthew During at the FAST 2014 Summit, now we have the exciting news that this sponsored clinical trial is moving forward. “We very much look forward to moving OV101 into the clinic, and hopefully making a […]