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 […]
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 […]
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 […]
[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 US and FAST AU Announce Joint Funding To Dr. Arthur Beaudet To Generate Mouse Lines Expressing Human UBE3A Antisense
“Of all the childhood genetic neurologic disorders of the brain, Angelman syndrome may be the single best candidate for developing a definitive treatment (aka a cure).”~Dr. Arthur Beaudet The Foundation for Angelman Syndrome Therapeutics (FAST) US and FAST AU are excited to announce joint funding to Dr. Beaudet to generate mouse lines expressing human UBE3A […]
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.
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.
Agilis Biotherapeutics and Dr. Edwin Weeber take giant step forward toward a functional cure for Angelman Syndrome. For Further information, read the press release here.
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 […]