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Scientific Studies

Scientific Studies

As part of our mission at the NPKUA, we strive to keep the community informed with the latest advances in PKU research. We are committed to sharing recent scientific abstracts, posters, and studies relevant to the PKU community.

During the 2024 NPKUA Conference, industry and academic partners showcased their scientific research through their scientific posters displayed throughout the weekend. See the incredible work of some of the leading PKU researchers in the posters and abstracts below:

Safety and Efficacy of NGGT002 in Adults with PKU

A Novel Gene Editing Approach for Phenylketonuria

NPKUA 2024 Poster

PKU Mouse Models Demonstrate Potential of Genome Editing Technology (RNA Gene Writers)

Preclinical Data of SAR444836 AAV Gene Addition Treatment

Inhaled Phenylalanine Ammonia Lyase (PAL) Reduction of Blood Phe in PKU Mouse Models

Neural Basis for Emotional Control Difficulties in Individuals with PKU

Exploring the Landscape of PKU Education and Learning Needs of Genetics Trainees

Optimizing Clinical Outcomes with Patients with PKU or Caregivers

Update from PHEFREE

Almost 40 Years of Experience with Large Neutral Amino Acids in Denmark

Simple and Quick Urine Test for Blood Phe Monitoring for PKU