Investigators2

INVESTIGATORS

Pietro Fratta, MD, PhD

University College London

2021 Harrington UK Rare Disease

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

Pietro fratta online3
Angela russell2

Angela Russell, DPhil

University of Oxford

2021 Harrington UK Rare Disease

Small Molecule AhR Antagonists for the Treatment of Duchenne Muscular Dystrophy

Helen Waller-Evans, DPhil

Cardiff University

2021 Harrington UK Rare Disease

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

Helen waller evans online2
Wyatt yue online2

Wyatt Yue, PhD

Newcastle University

2021 Harrington UK Rare Disease

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

Haiyan Zhou, MD, PhD

University College London

2021 Harrington UK Rare Disease

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

Haiyan zhou2

Paul V. Fish, PhD

University College London

2020 ADDF-Harrington

Restoration of Healthy Wnt Signaling in the Neurovascular Unit Through Inhibition of Notul Carboxylesterase Activity: A Lead to Preclinical Candidate Program for Alzheimer's Disease

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