Baylor receives NIH funding to launch...

Molly Chiu

713-798-4710

Houston, TX - Oct 19, 2026

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Baylor College of Medicine will receive more than $6 million over five years from the National Institutes of Health to lead the new Peroxisomal Disorders Consortium in the Rare Diseases Clinical Research Network. This national, multicenter consortium brings together investigators, clinical centers and patient organizations with expertise in a group of rare genetic conditions caused by impaired peroxisome function. These disorders can affect the brain, vision, hearing, liver, bones and other organ systems, but their clinical progression remains incompletely understood.

Peroxisomes are organelles that play a key role in breaking down large lipids inside the cell and building lipids for the brain and nerves. While peroxisomal disorders cover a heterogenous group of multisystem disorders with a wide spectrum of disease severity, the disorders share common pathophysiologic mechanisms, which may allow for shared therapeutic approaches. Peroxisomal disorders are rare diseases, but improved newborn screening programs are increasing neonatal diagnoses. In many cases, early interventions can profoundly improve long-term quality of life.

This consortium will conduct a prospective natural-history study to determine how peroxisomal disorders change over time and identify reliable clinical and biochemical measures for future therapeutic trials. Investigators also will integrate whole-genome sequencing, RNA sequencing and other molecular approaches with longitudinal clinical data to better understand differences in disease severity and identify potential therapeutic pathways. The consortium also aims to build a coordinated national research infrastructure. Investigators will partner closely with affected individuals and families and train the next generation of researchers in peroxisomal disorders.

"Twenty years ago, when I was a pediatric resident at Baylor College of Medicine, I met a family living with a peroxisome biogenesis disorder. That experience stayed with me and helped shape the work I have pursued ever since," said principal investigator Dr. Michael Wangler, associate professor of molecular and human genetics at Baylor and medical geneticist at Texas Children's Hospital. "Through this consortium, we will establish clinical research centers across the United States and, for the first time, offer families the opportunity to participate in a prospective natural-history study designed to improve diagnosis, identify meaningful biomarkers and generate the data needed to develop and evaluate better therapies."

"The Department of Molecular and Human Genetics has a long and distinguished history of leading collaborative research networks focused on rare diseases," said Dr. Sandesh Nagamani, professor and vice chair of molecular and human genetics at Baylor and lead of the Administrative Core of the Peroxisomal Disorders Consortium. "We have been active members of multiple consortia within the NIH Rare Diseases Clinical Research Network, and we are currently a primary awardee for three RDCRN consortia: the Urea Cycle Disorders Consortium, the Brittle Bone Disorders Consortium, and the Rare Organic Acidemias Research Consortium. We are honored to add the Peroxisomal Disorders Consortium to that list and look forward to working under Dr. Wangler's leadership to advance clinical investigation and care for this rare group of multisystem disorders."

PDC is funded under grant number U54TR006478 as a collaboration between the National Center for Advancing Translational Sciences, the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Eye Institute.