Advancing Research and Care: Propionic Acidemia Conference 

Friday, July 11 – 4:00-7:30 PM

4:00-7:30 PM Registration, light dinner, parent panel and discussion, visit exhibitors

Saturday, July 12 – 8:30 AM-4:00 PM

8:30-9:00 AM Registration / Exhibits Open

9:00-9:15 AM Welcome/Introductions

9:15-10:00 AM PA Overview/Research Charles P. Venditti MD, PhD, NHGRI, NIH

10:00-10:30 AM Break – Meet other families and visit exhibitors

10:30-11:15 AM Metabolic insights into PA: pathological mechanisms and emerging therapeutic strategies, Guofang Zhang, PhD, Duke University

11:15-11:45 PM TBD

11:45-1:00 PM Lunch

1:00-1:15 PM Cardiovascular Pathology in PA Across Zygosity Type, Andres Hollenbeck, Cardiology Care for Children

1:15-1:30 PM Electrophysiological Abnormalities in PA: ECG Analysis Across Genotypes, Carla Leal, BCN, Medtech group Physense

1:30-1:45 PM Machine Learning-Based Electrocardiographic Analysis to Characterize the Cardiac Phenotype in PA, Carla Leal, BCN, Medtech group Physense

1:45-2:30 PM Developing Medicines to Treat Liver Stop Codon Disease, Nerissa C. Kreher, MD, CMO, alltrna

2:30-3:00 PM Break – Meet other families and visit exhibitors

3:00-3:45 PM mRNA-3927 for PA: Aggregate Interim Results of Ph 1/2 and Long-Term Extension Trials, Rosa Real, MD, Moderna

3:45-4:00 PM Wrap up

*Program subject to change

A limited number of no-cost Echocardiograms and EKGs for the patients and families with PA will be available. To schedule, contact Daniela at 717-925-8300.

Sponsorships available – contact paf@pafoundation.com for more information.

Recent PAF Newsletters

Summer 2024 PAF Newsletter Spring 2024 PAF Newsletter Fall 2023 PAF Newsletter
Dr. Charles P. Venditti, M.D., Ph.D
Research approaches to study propionic acidemia: lessons from patients and mice
Zineb Ammous, MD FACMG, PA 
Propionic Acidemia Overview
Elaine Wakefield, RDN, LP
Fueling the Body
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