Unexpected differences in the pharmacokinetics of N-acetyl-DL-leucine enantiomers after oral dosing and their clinical relevance.

by Grant C Churchill, Michael Strupp, Antony Galione, Frances M Platt
Published 2020-01-01

Differential response of the liver to bile acid treatment in a mouse model of Niemann-Pick disease type C [version 1; referees: 2 approved, 1 not approved]

by Elena-Raluca Nicoli, David Smith, Lauren Morris, Frances M. Platt
Published 2017-08-01

Differential response of the liver to bile acid treatment in a mouse model of Niemann-Pick disease type C [version 2; referees: 2 approved, 1 not approved]

by Elena-Raluca Nicoli, Mylene Huebecker, David Smith, Lauren Morris, Frances M. Platt
Published 2018-04-01

Beneficial effects of anti-inflammatory therapy in a mouse model of Niemann-Pick disease type C1

by David Smith, Kerri-Lee Wallom, Ian M. Williams, Mylvaganam Jeyakumar, Frances M. Platt
Published 2009-11-01

Critical role of iron in the pathogenesis of the murine gangliosidoses

by Mylvaganam Jeyakumar, Ian Williams, David A. Smith, Timothy M. Cox, Frances M. Platt
Published 2009-06-01

Metabolomic Studies of Lipid Storage Disorders, with Special Reference to Niemann-Pick Type C Disease: A Critical Review with Future Perspectives

by Benita Claire Percival, Miles Gibson, Philippe B. Wilson, Frances M. Platt, Martin Grootveld
Published 2020-04-01

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

by Ecem Kaya, David A. Smith, Claire Smith, Barry Boland, Michael Strupp, Frances M. Platt
Published 2020-04-01

Case Report: Ursodeoxycholic acid treatment in Niemann-Pick disease type C; clinical experience in four cases [version 1; referees: 2 approved]

by William R.H. Evans, Elena-Raluca Nicoli, Raymond Y. Wang, Nina Movsesyan, Frances M. Platt
Published 2017-08-01

Brain Pathology in Mucopolysaccharidoses (MPS) Patients with Neurological Forms

by Gustavo M. Viana, David A. Priestman, Frances M. Platt, Shaukat Khan, Shunji Tomatsu, Alexey V. Pshezhetsky
Published 2020-02-01

Increased glycosphingolipid levels in serum and aortae of apolipoprotein E gene knockout mice

by Brett Garner, David A. Priestman, Roland Stocker, David J. Harvey, Terry D. Butters, Frances M. Platt
Published 2002-02-01

Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann–Pick type C1 mice

by Sarah N.R. Pressey, David A. Smith, Andrew M.S. Wong, Frances M. Platt, Jonathan D. Cooper
Published 2012-03-01

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

by Ian M. Williams, Kerri-Lee Wallom, David A. Smith, Nada Al Eisa, Claire Smith, Frances M. Platt
Published 2014-07-01

Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease

by Ulrika Andersson, David Smith, Mylvaganam Jeyakumar, Terry D Butters, Mario Cortina Borja, Raymond A Dwek, Frances M Platt
Published 2004-08-01

Inhibition of the Niemann-Pick C1 protein is a conserved feature of multiple strains of pathogenic mycobacteria

by Yuzhe Weng, Dawn Shepherd, Yi Liu, Nitya Krishnan, Brian D. Robertson, Nick Platt, Gerald Larrouy-Maumus, Frances M. Platt
Published 2022-09-01

Intracellular sphingosine releases calcium from lysosomes

by Doris Höglinger, Per Haberkant, Auxiliadora Aguilera-Romero, Howard Riezman, Forbes D Porter, Frances M Platt, Antony Galione, Carsten Schultz
Published 2015-11-01

Acetylation turns leucine into a drug by membrane transporter switching

by Grant C. Churchill, Michael Strupp, Cailley Factor, Tatiana Bremova-Ertl, Mallory Factor, Marc C. Patterson, Frances M. Platt, Antony Galione
Published 2021-08-01

Glucosylceramide modulates membrane traffic along the endocytic pathway

by Dan J. Sillence, Vishwajeet Puri, David L. Marks, Terry D. Butters, Raymond A. Dwek, Richard E. Pagano, Frances M. Platt
Published 2002-11-01

Reduced sphingolipid hydrolase activities, substrate accumulation and ganglioside decline in Parkinson’s disease

by Mylene Huebecker, Elizabeth B. Moloney, Aarnoud C. van der Spoel, David A. Priestman, Ole Isacson, Penelope J. Hallett, Frances M. Platt
Published 2019-11-01

Correction to: Reduced sphingolipid hydrolase activities, substrate accumulation and ganglioside decline in Parkinson’s disease

by Mylene Huebecker, Elizabeth B. Moloney, Aarnoud C. van der Spoel, David A. Priestman, Ole Isacson, Penelope J. Hallett, Frances M. Platt
Published 2020-01-01

Altered Expression of Ganglioside Metabolizing Enzymes Results in GM3 Ganglioside Accumulation in Cerebellar Cells of a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis

by Aleksandra Somogyi, Anton Petcherski, Benedikt Beckert, Mylene Huebecker, David A. Priestman, Antje Banning, Susan L. Cotman, Frances M. Platt, Mika O. Ruonala, Ritva Tikkanen
Published 2018-02-01