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2018 Harrington Rare Disease Scholars

Ed Grabczyk, PhD

Ed Grabczyk, PhD

LSU Health Sciences Center New Orleans

Small Molecule Induced Skipping of MLH3 Exon Seven to Slow Progression of DNA Repeat Expansion Disorder

Xianxin Hua, MD, PhD

Xianxin Hua, MD, PhD

University of Pennsylvania Pereleman School of Medicine

Developing Conventional and Switchable Car T Cells to Treat Acute Myeloid Leukemia (AML) by Targeting a Cell Surface Aminopeptidase

Justin Ichida, PhD

Justin Ichida, PhD

University of Southern California

Validation of PIKFYVE Inhibition as a Therapeutic Target for ALS

Jeannie Lee, MD, PhD

Jeannie Lee, MD, PhD

Massachusetts General Hospital

Advancing a Drug Prototype to Treat Rett Syndrome

John Marshall, PhD

John Marshall, PhD

Brown University

Early-Phase Clinical Development of a Lead Cyclic-PDZ-Enhancer Drug for Angelman Syndrome

Xin Qi, PhD

Xin Qi, PhD

Case Western Reserve University

Identification of Mitochondrial Functional Enhancers as Candidate Therapeutics for Huntington’s Disease

Daniel Scoles, PhD

Daniel Scoles, PhD

University of Utah

Development of a Therapeutic for SCA2 and Other Rare Neurodegenerative Diseases

James Shayman, MD

James Shayman, MD

University of Michigan

Brain Penetrant Glucosylceramide Synthase Inhibitors For the Treatment of CNS Based Glycosphingolipidoses

Alessandra Biffi, MD

Alessandra Biffi, MD

Dana-Farber Cancer Institute

Hematopoietic Stem Cell Gene Therapy for Rescue of Primary and Secondary Pathogenic Mechanisms in Infantile Neuronal Ceroid Lipofuscinosis

The Harrington Project for Discovery & Development

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