Study with Jaguar Gene Therapy

What is the natural history and course of type 1 galactosemia?
Recruitment goal
100
participants
Launched in
2022
Status
Active
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Partner logo

Overview

This research will help improve clinical understanding of the natural history and disease course of type 1 galactosemia. Natural history is the collection of information about the usual course of development of a disease or condition in the absence of treatment. Researchers will focus on a number of factors, including understanding more about how symptoms present in different age ranges, whether any symptoms correlate with specific lab values, and other potential predictors of disease severity. They will also look at use of healthcare services and resources by patients and caregivers affected by galactosemia.

Study goal

The goal of this study is to understand the natural history of type 1 galactosemia.

Research partners

About AllStripes

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