The clinical research team in the Division of Genetics works on a variety of research studies. These include natural history studies which document the course of disorders before treatment and during treatment. We are also involved in Phase I (first in human) through Phase IV (post-FDA approval) clinical trials. The focus of these trials is to develop new therapies and improve existing therapies. Genetic disorders that are studied include:
Phenylketonuria (PKU), and other amino acid disorders
Fatty acid oxidation disorders
Urea cycle disorders
Lysosomal storage disorders
Metachromatic leukodystrophy (MLD)
Questions or inquiries for any of the clinical research trials can be directed to Rachel Katz, Clinical Research Manager, at RKatz@luriechildrens.org.