Neonatal Screening Articles & Analysis
6 news found
Clinical data demonstrating initial safety and efficacy from the RESKUE trial are being presented by Chief Medical Officer Maria Escolar, M.D., at the 29th Congress of European Society of Gene & Cell Therapy (ESGCT), October 11-14, 2022 Subjects treated with FBX-101 have shown increased galactocerebrosidase (GALC) enzyme activity in plasma and cerebrospinal fluid (CSF), normal white matter ...
FBX-101 clinical data demonstrating safety and initial efficacy from the RESKUE trial is being presented at the 2022 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium FBX-101 is a systemic AAV gene therapy following unrelated cord blood transplantation (“UCBT”) and has been well tolerated through Day 180 FBX-101 significantly increased GALC enzyme ...
HemoShear Therapeutics, Inc., a clinical stage company developing treatments for rare metabolic disorders, today announced that the first two patients have been dosed in the HERO (HElp Reduce Organic Acids) Phase 2 clinical trial of HST5040, an oral small molecule drug being investigated for the treatment of patients with methylmalonic acidemia (MMA) and propionic acidemia (PA). This trial seeks ...
Evolve BioSystems, Inc. today announces that its product, activated B. infantis EVC001, will be used in one of the largest international clinical studies on preventing type 1 diabetes (T1D) in genetically predisposed children. The randomized, controlled, double-blind, multicenter trial will be conducted across eight major research centers in five European countries. The Leona M. and Harry B. ...
Hollywood, FL - Abbott Informatics is pleased to announce the launch of the STARLIMS Public Health Solution PH12.0. Abbott Informatics has the STARLIMS Public Health (PH) Solution product currently deployed in laboratories throughout the United States. Nevertheless, enhancements need to continuously be added in order for our product to satisfy evolving market needs. Newborn screening is one such ...
The award will enable Dr Yogesan Kanagasingam, Research Director for the Australian e-Health Research Centre (AEHRC), to spend four months at Stanford University School of Medicine developing and validating a new ‘non-invasive’ system to screen newborn babies. CSIRO Chief Executive, Dr Megan Clark, said Dr Kanagasingam’s work has application across a wide range of areas, ranging from remote, ...