The Emory Genetic Clinical Trials Center (GCTC) consists of international experts focused on treating children with genetic diseases, defined by leading standards of care, pioneering protocols, and individualized services in a world-class medical environment. Our team’s successes result from the combined clinical and research expertise of our medical geneticists, scientists, developmental pediatricians, genetic counselors, psychologists, metabolic dietitians, and experienced clinical research coordinators and nurses.
The center is focused on novel therapeutic modalities and personalized services to treat infants, children, adolescents, and young adults who are diagnosed with the following groups of inherited conditions:
- Lysosomal Storage Diseases including: Fabry disease, Gaucher disease, Pompe disease, Mucopolysacchridosis Type I (Hurler, Hurler Scheie, Scheie), Mucopolysaccharidosis Type II (Hunter syndrome), Mucopolysaccharidosis Type IV (Morquio), Neimann-Pick (A, B, and C), Lysosomal Acid Lipase Deficiency, and others.
- Neurocognitive Conditions including: Fragile X, Down Syndrome (Trisomy 21), autism, and others.
- Inborn Errors of Metabolism including: Phenylketonuria (PKU), Urea cycle disorders, and others.
- Skeletal Dysplasias including: Achondroplasia, Hypophosphastia, Osteogenesis Imperfecta, and others.
- Other Genetic Conditions such as Cerebrotendinous xanthomatosis (CTX)
In addition to conducting the clinical trials, behind the scenes, the studies are organized by highly-trained professional genetic counselors, clinical coordinators, and research personnel specializing in areas of clinical coordination, data management, specimen processing and regulatory management, Emory Genetic Clinical Trials Center provides a supportive environment to conduct clinical trials in a cost-effective and efficient manner while ensuring compliance with Emory clinical trials standard operating procedures, Good Clinical Practice (GCP), Emory University Institutional Review Board (IRB), US Food and Drug Administration (FDA), other regulatory agencies and external sponsors.
To learn more about our clinical trials please contact our GCTC Team at lsd-gctc-l@listserv.cc.emory.edu or 404-778-8518.