RARE - Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting | Benzinga
NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that preclinical and clinical data, including new clinical efficacy and safety data from the expansion cohorts in the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome, will be presented at the 76th Annual American Academy of Neurology Meeting (AAN) being held April 13-18 in Denver and virtually. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS).
Abstracts are available to view online at www.aan.com/events/annual-meeting-abstracts. The GTX-102 emerging science presentation will include data beyond what is currently available online.
AAN 2024 Presentation Details:
Title: Clinical Activity and Safety of GTX-102, an Investigational Antisense Oligonucleotide for the Treatment of Patients With Angelman Syndrome
Format: Oral (abstract #192, presentation #008)
Session: Emerging Science 2
Presenter: Kemi A. Olugemo, M.D., FAAN, Ultragenyx
Date/ Time: Tuesday, April 16, 6:12-6:18 p.m. MDT
Title: Proteomic Profiling of Angelman Syndrome for Disease-Associated Biomarker Discovery
Format: Poster (#8.001)
Session: Child Neurology and Developmental Neurology: Neurogenetics 2
Presenter: Rachael Elizabeth Hawtin, Ph.D., Ultragenyx
Date/ Time: Tuesday, April 16, 5:30-6:30 p.m. MDT
Title: Clinical Characteristics of Creatine Transporter Deficiency (CTD): Final Results of the Vigilan Observational Study
Format: Poster (#8.002)
Session: Child Neurology and Developmental Neurology: Neurogenetics 2
Presenter: Melanie Brandabur, M.D., Ultragenyx
Date/ Time: Tuesday, April 16, 5:30-6:30 p.m. MDT
Conference Call and Webcast Information
Ultragenyx will host a conference call at 8:00 a.m. ET on Wednesday, April 17, 2024, to ...