GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome
SARASOTA, Fla. and NOVATO, Calif., Oct. 26, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - October 28, 2020 Category: Pharmaceuticals Source Type: clinical trials
Angelman Syndrome Natural History Study
Condition: Angelman Syndrome Intervention: Sponsors: Boston Children’s Hospital; Food and Drug Administration (FDA); University of California, San Diego Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - August 11, 2020 Category: Research Source Type: clinical trials
A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome
Condition: Angelman Syndrome Intervention: Drug: RO7248824 Sponsor: Hoffmann-La Roche Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - June 12, 2020 Category: Research Source Type: clinical trials