Applied Therapeutics to Present Data on AT-007 for Treatment of Galactosemia at the Galactosemia Foundation 2020 Virtual Conference
“We are excited to share our development progress with the Galactosemia community,” said
Oral Presentation Details
Title: AT-007: Development of an Oral Treatment for Patients with Galactosemia
Title: ACTION-Galactosemia: Clinical Experience with Adult Galactosemia Patients and Path Forward
Title: ACTION-Galactosemia Kids: Pediatric Study of AT-007 in Children with Galactosemia
Slides will be available on the presentations and publications section of the
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in clinical development for treatment of Galactosemia. AT-007 has been studied in an animal model of Galactosemia, which demonstrated that AT-007 reduces toxic galactitol levels and prevents disease complications. Applied Therapeutics is conducting a biomarker based development program in patients with Galactosemia, based on the recently released industry guidance on drug development for low prevalence, slowly progressing rare metabolic diseases. The company received Orphan Designation for AT-007 for Galactosemia in May 2019 and Pediatric Rare Disease Voucher (PRV) designation in 2020.
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Source: Applied Therapeutics