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Publications by Frances M. Platt

Publisher Correction: Lysosomal Storage Diseases

Nature Reviews Disease Primers

English

Correction To: Reduced Sphingolipid Hydrolase Activities, Substrate Accumulation and Ganglioside Decline in Parkinson’s Disease

Molecular Neurodegeneration

Molecular Neuroscience

Molecular Biology

English

Biomarkers for Disease Progression and AAV Therapeutic Efficacy in Feline Sandhoff Disease

Experimental Neurology

Developmental Neuroscience

English

Mechanistic Convergence and Shared Therapeutic Targets in Niemann‐Pick Disease Type C and Tangier Disease

Journal of Inherited Metabolic Disease

English

The Yeast P5 Type ATPase, Spf1, Regulates Manganese Transport Into the Endoplasmic Reticulum

Multidisciplinary

English

Synthesis and Study of Novel Multifunctional Cyclodextrin‐Deferasirox Hybrids

Organic Chemistry

Molecular Medicine

English

NPC1 Deficiency in Mice Is Associated With Fetal Growth Restriction, Neonatal Lethality and Abnormal Lung Pathology

Journal of Clinical Medicine

English

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

Journal of Clinical Medicine

English

Extensive Glycosphingolipid Depletion in the Liver and Lymphoid Organs of Mice Treated withN-Butyldeoxynojirimycin

Journal of Biological Chemistry

Molecular Biology

English