Genetic fingerprint sheet

2021 Harrington UK Rare Disease Scholars

Pietro Fratta

Pietro Fratta, MD, PhD

University College London

A Muscle Targeted Gene Therapy for Kennedy’s Disease, a Rare Neuromuscular Disease

Angela Russell, DPhil

Angela Russell, DPhil

University of Oxford

Small Molecule AhR Antagonists for the Treatment of Duchenne Muscular Dystrophy

Helen Waller-Evans, DPhil

Helen Waller-Evans, DPhil

Cardiff University

Inhibitors of Acid Ceramidase to Treat Multiple Lysosomal Storage Disorders, Causes of Widespread Organ Damage

Wyatt Yue

Wyatt Yue, PhD

University of Oxford

Hit-to-Lead Development of HAO1 Inhibitors of Primary Hyperoxaluria, A Cause of Kidney Failure

Haiyan Zhou, MD, PhD

Haiyan Zhou, MD, PhD

University College London

Developing Antisense Oligonucleotide Therapy for SPTLC1-Related Hereditary Sensory Neuropathy, A Cause of Shooting Pain

The Harrington Project for Discovery & Development

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