Results for Leukodystrophy

Using translational approaches that encompass genomic studies, biomarker development, disease modeling, natural history studies, and clinical trials, Dr. Vanderver seeks to improve the quality of life and lifespan of individuals living with leukodystrophies or heritable disorder of myelin. She leads the Leukodystrophy Center of Excellence at Children's Hospital of Philadelphia.

Dr. Shults works to develop statistical methods for longitudinal data that include semi-parametric approaches to account for subject/cluster level associations and maximum likelihood-based approaches for simulation and analysis of discrete longitudinal outcomes that may have overdispersion.

Dr. Waldman works to understand metrics to quantify disease burden in acquired and genetic neurologic disorders that affect the white matter of the brain. She also focuses on clinical trial methodology to bring novel therapies to neurodegenerative diseases.

Dr. Thomas is a pediatric neuropsychologist with research interests in the neuropsychological effects of medical conditions including leukodystrophies and other neurologic disease, pediatric sepsis, neurodermatoses, cardiac conditions, genetic disorders, and rheumatic and endocrine disorders.

Dr. Baldwin is an orthopaedic surgeon who specializes in the treatment of traumatic, neuromuscular, and spinal conditions in children.

Dr. Tricoli's research involves developing a new generation of lentiviral vectors that will prove to limit toxicity and improve activity of the ARSA enzyme, extending the efficacy of and eligibility for gene therapy in metachromatic leukodystrophy, a devastating childhood lysosomal storage disease.