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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

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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

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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

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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

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Interview Study Seeks Caregivers of Children/Adolescents with Angelman Syndrome.

The purpose of this interview study is to gather information about caregivers’ experiences with Angelman syndrome (AS). Caregivers of patients aged 5 to 17 years will be interviewed about their experiences with AS. Caregivers will also be asked to complete, and evaluate, a number of questionnaires in terms of their relevance to AS patients, appropriateness,

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Genomic Imprinting Does Not Reduce the Dosage of UBE3A in Neurons

Summary by Allyson Berent, DVM, DACVIM Angelman syndrome (AS) is caused by loss of the maternally inherited copy of the UBE3A gene. Like most genes, the UBE3A gene is present on both alleles of chromosome 15, the one inherited from the mother (maternal allele) and the one inherited from the father (paternal allele); however, UBE3A

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Quantitative Measurement of Communication Ability in Children with Angelman Syndrome

Summary by Allyson Berent, DVM, DACVIM Individuals affected with Angelman syndrome (AS) are known to have limited functional speech and are typically able to communicate through gestures, sounds, minimal signs, and augmentative communication devices (AAC). The lack of functional speech in AS is thought to be associated with cognitive delays and oral motor dysfunction. Traditional

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Private Dinner with Colin Farrell and Dr. Edwin Weeber

Starting today, February 1, 2017 and running through midnight CST on June 30, 2017, you and a guest have the chance to win an all-expense paid trip* to Los Angeles, California, for a private dinner with critically acclaimed actor, Golden Globe winner and fellow Angelman parent Colin Farrell and world-renowned scientist Dr. Edwin Weeber! As

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Ganaxalone As Potential Therapeutic For Angelman Syndrome

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 tolerance did not occur to the therapeutic effects of the drug. The results support clinical studies to investigate

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Gene Therapy in Angelman Syndrome: Agilis Biotherapeutics

By Allyson Berent, DVM, DACVIM  At the annual Foundation for Angelman Syndrome Therapeutics (FAST) Global Summit in December, Jodi Cook, Ph.D., Chief Operating Officer for Agilis Biotherapeutics (Agilis), presented on gene therapy development for Angelman Syndrome (AS). Her presentation was very exciting for the community as we prepare to bring gene therapy treatment into human

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