Claire Smith

Improved neuroprotection using miglustat, curcumin and ibuprofen as a triple combination therapy in Niemann–Pick disease type C1 mice

F. Platt, Kerri-Lee Wallom, David A Smith, 2014, Neurobiology of Disease.

Beneficial Effects of Acetyl-DL-Leucine (ADLL) in a Mouse Model of Sandhoff Disease

M. Strupp, F. Platt, David A Smith, 2020, Journal of clinical medicine.

Defective iron homeostasis and hematological abnormalities in Niemann-Pick disease type C1

F. Platt, F. Porter, A. Abdul-Sada, 2022, Wellcome open research.

Heat shock protein amplification improves cerebellar myelination in the Npc1nih mouse model

F. Platt, T. Kirkegaard, David A Smith, 2022, EBioMedicine.

Imaging of changes in copper trafficking and redistribution in a mouse model of Niemann-Pick C disease using positron emission tomography

J. Spencer, F. Platt, R. Purchase, 2019, BioMetals.

Acetyl-leucine slows disease progression in lysosomal storage disorders

M. Strupp, J. Spencer, M. Cortina-Borja, 2020, bioRxiv.

A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease

A. Rahim, David A Smith, Claire Smith, 2023, Cells.