Blog

SAB Meet and Greet: Welcome Dr. Duis

Barb Bailus, PhD FAST is always on the lookout for excellent physicians who interact with AS patients and can help design and explain what is feasible in a clinical trial. Dr. Jessica Duis is one such physician, and is currently the medical director of the Angelman Syndrome comprehensive clinic at Vanderbilt Children’s Hospital in Tennessee. Dr.

Read More >

SAB Meet and Greet: Welcome Dr. Zylka

Barb Bailus, PhD Dr. Mark Zylka is so new to FAST that he hasn’t yet joined us at a FAST Gala, but that is something that should change this year. He did listen to all 11 hours of the FAST Science Summit in 2018, which inspired him to work more closely with the mission of

Read More >

SAB Meet and Greet: Welcome Dr. Beaudet

Barb Bailus, PhD Our next new scientific advisory board (SAB) member could be considered the grandfather of Angelman syndrome (AS), Dr. Art Beaudet. Dr. Beaudet received his MD from Yale and did his pediatric residency at Johns Hopkins before joining Baylor College of Medicine, where he has resided since 1971. Dr.Beaudet first became interested in AS

Read More >

SAB Meet and Greet: Welcome Dr. Yu

Barb Bailus, PhD Today we start a series of posts to introduce our newest FAST Scientific Advisory Board members; it is an honor to welcome such an amazing group of scientists! Several of these scientists have been members of the Angelman community for a long time, and others are fairly new but offer exciting insights

Read More >

Angels in the City – Celebrating Angelman research in Los Angeles

By Lauren Hoffer Getting out of town is hard.  I don’t need to tell any of you this.  The day before we left, Greg looked at me and said, “Today is going to be painful.”  We had fast and furious workdays squaring things away.   We did the usual parenting gig; “keeping the babies alive” we

Read More >

FAST Welcomes New Chief Scientific Communications Reporter, Barb Bailus PhD

Understand the Power of Your Donations Hello fellow Angelman Community, as this is my first “official” blog I am going to take a moment and introduce myself.  I am a research scientist with a specific focus on genetic and neurological disorders.  As a doctoral student I did my research in Dr. David Segal’s lab, investigating

Read More >

GeneTx Biotherapeutics receives FDA orphan-drug designation for Angelman syndrome treatment

Downers Grove, Ill., March 27, 2018 – The U.S. Food and Drug Administration has granted orphan-drug designation to GeneTx Biotherapeutics LLC’s GTX-101 for the treatment of Angelman syndrome, a rare neurogenetic disorder that affects approximately one in 15,000 people. GTX-101 is the first drug candidate for the startup biotechnology company. “No approved treatments for Angelman

Read More >

New biotech launched for antisense program targeting Angelman syndrome

Downers Grove, Ill., Feb. 22, 2018 – FAST (Foundation for Angelman Syndrome Therapeutics) announced today the launch of GeneTx Biotherapeutics LLC (GeneTx), a subsidiary of FAST, for the development and commercialization of an investigational antisense drug, GTX-101, for the treatment of Angelman syndrome (AS). FAST selected Paula Evans, the chairperson of FAST, to serve as

Read More >

About GeneTx

Beginning in early 2012, FAST began funding the Dindot laboratory at Texas A&M University to understand the mechanism by which UBE3A-AS turns off the paternal UBE3A allele, why the paternal UBE3A allele is off in neurons, and how that process may be circumvented as a potential therapy for AS. Research in the Dindot laboratory identified

Read More >

^