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
AllStripes is a platform that makes it easy for rare disease patients and caregivers to contribute to drug development research from home.