Frances M Platt Selected Research

Lysosomal Storage Diseases (Lysosomal Storage Disease)

1/2022	Vesicle cholesterol controls exocytotic fusion pore.
1/2021	Acetyl-leucine slows disease progression in lysosomal storage disorders.
4/2020	Beneficial Effects of Acetyl-DL-Leucine (ADLL) in a Mouse Model of Sandhoff Disease.
1/2020	Upregulating β-hexosaminidase activity in rodents prevents α-synuclein lipid associations and protects dopaminergic neurons from α-synuclein-mediated neurotoxicity.
11/2016	Pathogenic mycobacteria achieve cellular persistence by inhibiting the Niemann-Pick Type C disease cellular pathway.
1/2016	Urinary excretion and metabolism of miglustat and valproate in patients with Niemann-Pick type C1 disease: One- and two-dimensional solution-state (1)H NMR studies.
1/2016	Immune dysfunction in Niemann-Pick disease type C.
2/2014	RIPK3 as a potential therapeutic target for Gaucher's disease.
3/2012	Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann-Pick type C1 mice.
8/2011	Lysosomal Ca(2+) homeostasis: role in pathogenesis of lysosomal storage diseases.

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Frances M Platt Research Topics

Disease

27	Type C Niemann-Pick Disease (Niemann Pick Disease, Type C) 01/2022 - 08/2004
17	Lysosomal Storage Diseases (Lysosomal Storage Disease) 01/2022 - 05/2004
14	Sandhoff Disease (Sandhoff's Disease) 01/2021 - 08/2002
13	Neurodegenerative Diseases (Neurodegenerative Disease) 01/2022 - 08/2003
11	Gaucher Disease (Gaucher's Disease) 01/2020 - 01/2002
6	Disease Progression 01/2021 - 11/2004
6	GM2 Gangliosidoses (GM2 Gangliosidosis) 01/2021 - 08/2002
6	Infertility (Sterility) 12/2007 - 12/2002
5	Inflammation (Inflammations) 01/2022 - 11/2004
5	GM1 Gangliosidosis (Gangliosidosis GM1) 01/2021 - 05/2004
5	Gangliosidoses (Gangliosidosis) 01/2017 - 08/2003
3	Parkinson Disease (Parkinson's Disease) 01/2022 - 11/2019
3	Nervous System Diseases (Neurological Disorders) 01/2021 - 06/2009
3	Sphingolipidoses (Sphingolipidosis) 04/2020 - 11/2002
3	Neuroinflammatory Diseases 02/2020 - 07/2014
3	Metabolic Diseases (Metabolic Disease) 01/2018 - 06/2010
3	Tay-Sachs Disease 01/2018 - 01/2015
3	Infections 11/2016 - 05/2006
3	Fabry Disease (Fabry's Disease) 01/2016 - 06/2010
3	Body Weight (Weight, Body) 07/2014 - 05/2004
3	Male Infertility (Male Sterility) 06/2008 - 04/2005
2	Mucopolysaccharidoses 01/2022 - 02/2020
2	Memory Disorders (Memory Loss) 01/2022 - 01/2017
2	Crohn Disease (Crohn's Disease) 01/2022 - 01/2017
2	Ataxia (Dyssynergia) 01/2021 - 04/2020
2	Gliosis 02/2020 - 01/2017
2	Amyotrophic Lateral Sclerosis (Lou Gehrig's Disease) 12/2017 - 12/2015
2	Liver Diseases (Liver Disease) 01/2017 - 10/2016
2	Mucolipidoses (Sialidosis) 09/2014 - 08/2011
2	Alzheimer Disease (Alzheimer's Disease) 11/2010 - 07/2008
2	Congenital Abnormalities (Deformity) 12/2007 - 04/2005
2	Neoplasms (Cancer) 04/2007 - 09/2004
2	Teratozoospermia 01/2007 - 04/2005
2	Atherosclerosis 09/2004 - 02/2002
2	Hepatitis B 02/2004 - 01/2002
1	Mucopolysaccharidosis III (Sanfilippo Syndrome) 01/2022
1	Inflammatory Bowel Diseases (Inflammatory Bowel Disease) 01/2022
1	Acute-Phase Reaction 12/2021
1	Breast Neoplasms (Breast Cancer) 09/2021
1	Osteoporosis 09/2021
1	Vertigo (Positional Vertigo) 01/2021
1	Hereditary Sensory and Autonomic Neuropathies (HSAN) 01/2021
1	Cerebellar Ataxia (Dysmetria) 04/2020
1	Tangier Disease 01/2020
1	Hemosiderosis 01/2019
1	Bacterial Infections (Bacterial Infection) 01/2019

Drug/Important Bio-Agent (IBA)

20	GlycosphingolipidsIBA 01/2022 - 11/2002
15	EnzymesIBA 01/2021 - 01/2002
15	miglustat (Zavesca)FDA Link 01/2020 - 08/2003
14	LipidsIBA 01/2022 - 08/2004
13	GangliosidesIBA 01/2020 - 08/2003
12	CholesterolIBA 01/2022 - 11/2009
9	Pharmaceutical PreparationsIBA 01/2021 - 05/2004
9	SphingolipidsIBA 01/2020 - 11/2002
9	CalciumIBA 11/2016 - 08/2003
8	Proteins (Proteins, Gene)FDA Link 02/2020 - 07/2008
6	Glucosylceramidase (Glucocerebrosidase)IBA 01/2022 - 11/2002
6	Biomarkers (Surrogate Marker)IBA 11/2021 - 02/2009
6	G(M2) Ganglioside (Ganglioside GM2)IBA 04/2020 - 08/2003
6	Imino SugarsIBA 12/2007 - 01/2002
5	SugarsIBA 06/2010 - 11/2002
4	Lysergic Acid Diethylamide (LSD)IBA 01/2022 - 08/2011
4	migalastatIBA 05/2008 - 05/2004
3	Glucosylceramides (Glucocerebrosides)IBA 12/2017 - 11/2002
3	GlucosidasesIBA 01/2017 - 08/2004
2	Heparitin Sulfate (Heparan Sulfate)IBA 01/2022 - 02/2020
2	GalactosidasesIBA 01/2021 - 09/2012
2	acetylleucineIBA 01/2021 - 04/2020
2	Leucine (L-Leucine)FDA Link 01/2021 - 01/2021
2	HydrolasesIBA 01/2020 - 11/2019
2	Hexosaminidases (Hexosaminidase)IBA 01/2020 - 08/2002
2	AntigensIBA 11/2019 - 10/2008
2	G(M3) GangliosideIBA 02/2018 - 01/2017
2	CeramidesIBA 12/2017 - 08/2004
2	SphingosineIBA 01/2016 - 11/2008
2	ceramide glucosyltransferaseIBA 12/2015 - 09/2004
2	Ibuprofen (Motrin)FDA LinkGeneric 07/2014 - 11/2004
2	lysosomal proteinsIBA 07/2010 - 08/2003
2	beta-N-AcetylhexosaminidasesIBA 06/2010 - 05/2008
2	GlucosyltransferasesIBA 06/2010 - 08/2004
1	Glucosamine (Dona)IBA 01/2022
1	Acetyl Coenzyme A (Acetyl-CoA)IBA 01/2022
1	AcetyltransferasesIBA 01/2022
1	Fatty Acids (Saturated Fatty Acids)IBA 12/2021
1	Selective Estrogen Receptor Modulators (SERM)IBA 09/2021
1	Serine C-PalmitoyltransferaseIBA 01/2021
1	Neuroprotective AgentsIBA 01/2021
1	1-deoxysphingolipidIBA 01/2021
1	Keratan SulfateIBA 02/2020
1	sialogangliosidesIBA 02/2020
1	GlycosaminoglycansIBA 02/2020
1	Synapsins (Synapsin I)IBA 01/2020
1	Benzoic Acid (Potassium Benzoate)FDA Link 01/2019
1	Deferasirox FDA Link 01/2019

Therapy/Procedure

20	Therapeutics 01/2022 - 01/2003
4	Enzyme Replacement Therapy 01/2018 - 04/2008
4	Oral Administration 01/2016 - 12/2002
2	Contraception (Birth Control) 12/2007 - 12/2002
1	Off-Label Use 01/2021
1	Treatment Delay 01/2021