Mapping the Future for Liver Health: Metabolic and Genetic Disease Discoveries

Description

This session covers advances in inherited metabolic liver diseases — Wilson disease, alpha-1 antitrypsin deficiency, and acute hepatic porphyria — including natural history data and emerging targeted therapies.

Presentations

11:00 AM - 11:15 AM
Convention Center - Mile High Ballroom 2B-3B
Recorded session

Beyond Cirrhosis: Major Adverse Liver Outcomes Across PiMZ, PiSZ, and PiZZ Genotypes in Alpha-1 Antitrypsin Deficiency 

Dustin Bastaich, PhD | Abstract Presenter
11:15 AM - 11:30 AM
Convention Center - Mile High Ballroom 2B-3B
Recorded session

Atp7b inactivation disrupts hepatic metabolic zonation and drives diet-dependent steatohepatitis in a mouse model of Wilson Disease. 

Mugagga Kalyesubula | Abstract Presenter
11:30 AM - 11:45 AM
Convention Center - Mile High Ballroom 2B-3B
Recorded session

Defining the hepatic secretome in Wilson Disease using a lipid nanoparticle gene delivery system for proximity biotinylation of secreted proteins

Robert M Hughes, MD, PhD | Abstract Presenter
11:45 AM - 12:00 PM
Convention Center - Mile High Ballroom 2B-3B
Recorded session

Real World Evidence of Therapy Impact in Unstable Wilson Disease using Non-Ceruloplasmin Bound Copper by Speciation as a Novel Monitoring Tool 

Michael L. Schilsky, MD, FAASLD | Abstract Presenter
12:00 PM - 12:15 PM
Convention Center - Mile High Ballroom 2B-3B
Recorded session

Analysis of Acute Hepatic Porphyria Metabolite (ALA/PBG) Fluctuations and Attack Rate With Givosiran Treatment in the Phase 3 ENVISION Study

Bruce Wang, MD | Abstract Presenter
12:15 PM - 12:30 PM
Convention Center - Mile High Ballroom 2B-3B
Recorded session

Residual Hepatic, Neurologic, and Psychiatric Disease Burden in Treatment-Experienced Patients with Wilson Disease: Baseline Findings from the FoCus Phase 3 Trial

Frederick Askari, MD, PhD | Abstract Presenter

Objectives

  • Describe the spectrum of disease caused by alpha-1 antitrypsin deficiency based on phenotype
  • List the potential mechanisms of hepatic injury in Wilson's disease
  • Evaluate new methods of diagnosing and monitoring disease progression and treatment in patients with Wilson's disease
  • Evaluate new therapies proposed for acute hepatic porphyria