Frances M. Platt

Mansfield Road
Oxford, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 153 publications

Behind the photographs: Close relations between German pharmacologists and the British Pharmacological Society in the 1930s observed through the diary of Wolfgang Heubner.

Behind the photographs: Close relations between German pharmacologists and the British Pharmacological Society in the 1930s observed through the diary of Wolfgang Heubner.

Journal: British journal of pharmacology

Published: September 07, 2026

A Contemporary Pathomechanistic Nosology of Inherited Lysosomal Disorders.

A Contemporary Pathomechanistic Nosology of Inherited Lysosomal Disorders.

Journal: Journal of inherited metabolic disease

Published: July 01, 2026

HKDC1 contributes to aberrant lysosome-mitochondria contact in Niemann-Pick disease type C.

HKDC1 contributes to aberrant lysosome-mitochondria contact in Niemann-Pick disease type C.

Journal: bioRxiv : the preprint server for biology

Published: June 12, 2026

Erratum to "Links between COVID-19, long COVID, and neurodegeneration: The role of glycosphingolipids" [Pharmacological Reviews 78 (2026) 100113].

Erratum to "Links between COVID-19, long COVID, and neurodegeneration: The role of glycosphingolipids" [Pharmacological Reviews 78 (2026) 100113].

Journal: Pharmacological reviews

Published: May 20, 2026

N-acetyl-L-leucine normalizes Transcription Factor EB activity by stereospecific bidirectional modulation in a HeLa cell model of Niemann-Pick disease type C.

N-acetyl-L-leucine normalizes Transcription Factor EB activity by stereospecific bidirectional modulation in a HeLa cell model of Niemann-Pick disease type C.

Journal: PloS one

Published: March 20, 2026

Correction: Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N‑of‑1 Trial with 21 months Clinical Observation.

Correction: Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N‑of‑1 Trial with 21 months Clinical Observation.

Journal: Neurology and therapy

Published: March 12, 2026

Synthesis of N-Acetyl-D- and -L-Leucine-13C6 Tool Compounds in Neurodegenerative Disease.

Synthesis of N-Acetyl-D- and -L-Leucine-13C6 Tool Compounds in Neurodegenerative Disease.

Journal: ChemMedChem

Published: February 26, 2026

Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration.

Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration.

Journal: Human gene therapy

Published: December 29, 2025

Correction: Glycoprotein non-metastatic melanoma protein B is a biomarker of inflammation in individuals with Gaucher disease: relationship to clinico-pathological subtypes.

Correction: Glycoprotein non-metastatic melanoma protein B is a biomarker of inflammation in individuals with Gaucher disease: relationship to clinico-pathological subtypes.

Journal: Orphanet journal of rare diseases

Published: November 18, 2025

Molecular and biochemical insights into dysregulation of glycosphingolipid metabolism in a mouse model of lysosomal free sialic acid storage disorder.

Molecular and biochemical insights into dysregulation of glycosphingolipid metabolism in a mouse model of lysosomal free sialic acid storage disorder.

Journal: Experimental neurology

Published: November 05, 2025

2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C.

2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C.

Journal: Journal of inherited metabolic disease

Published: October 31, 2025

Limited therapeutic efficacy of N-acetyl-L-leucine in a mouse model of CLN1 disease.

Limited therapeutic efficacy of N-acetyl-L-leucine in a mouse model of CLN1 disease.

Journal: Scientific reports

Published: September 23, 2025

Showing 1-12 of 153

Last Updated: 09/26/2026