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M. Frascella · SciRah

Researcher profile · Semantic Scholar

M. Frascella

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M. Frascella — researcher profile on SciRah

h-index: 9
Works: 27
Citations: 540

Most-cited papers

The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease.
Richie Khanna, R. Soska, Lun Yi, Jessie Feng +9 more
2010Molecular Therapy138 citations Open access
Semantic Scholar
Co-administration With the Pharmacological Chaperone AT1001 Increases Recombinant Human α-Galactosidase A Tissue Uptake and Improves Substrate Reduction in Fabry Mice
Elfrida R. Benjamin, Richie Khanna, A. Schilling, J. Flanagan +12 more
2012Molecular Therapy96 citations Open access
Semantic Scholar
The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease
Richie Khanna, J. Flanagan, Jessie Feng, R. Soska +6 more
2012PLoS ONE83 citations Open access
Semantic Scholar
Improved efficacy of a next-generation ERT in murine Pompe disease.
Su Xu, Lun Yi, M. Frascella, Anadina Garcia +15 more
2019JCI Insight79 citations Open access
Semantic Scholar
Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice
Su Xu, Lun Yi, N. Brignol, R. Hamler +21 more
2015Molecular Therapy37 citations Open access
Semantic Scholar
The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse Model of Pompe Disease
Richie Khanna, A. Powe, Lun Yi, R. Soska +12 more
2014PLoS ONE37 citations Open access
Semantic Scholar
Glucosylceramide and Glucosylsphingosine Quantitation by Liquid Chromatography-Tandem Mass Spectrometry to Enable In Vivo Preclinical Studies of Neuronopathic Gaucher Disease.
R. Hamler, N. Brignol, S. Clark, Sean Morrison +7 more
2017Analytical Chemistry27 citations
Semantic Scholar
Discovery of Potent and Selective Inhibitors of Wild-Type and Gatekeeper Mutant Fibroblast Growth Factor Receptor (FGFR) 2/3.
Artem Shvartsbart, Jeremy J. Roach, Michael R. Witten, H. Koblish +22 more
2022Journal of Medicinal Chemistry20 citations Open access
Semantic Scholar
Discovery of Orally Bioavailable FGFR2/FGFR3 Dual Inhibitors via Structure-Guided Scaffold Repurposing Approach.
Minh-Hai Nguyen, Hai-Fen Ye, Yao Xu, L. Truong +22 more
2023ACS Medicinal Chemistry Letters16 citations Open access
Semantic Scholar
Novel recombinant human acid α-glucosidase with optimal glycosylation is significantly better than standard of care enzyme replacement for glycogen clearance in skeletal muscles of GAA knock-out mice
R. Gotschall, Su Xu, Lun Yi, A. Schilling +9 more
20155 citations
Semantic Scholar
Exploring the Use of Pharmacological Chaperone AT3375 Alone and in Combination with Recombinant human ß-Glucosidase for Gaucher Disease
Richie Khanna, Lee J. Pellegrino, R. Soska, Lun Yi +9 more
20123 citations
Semantic Scholar
METABOLIC MYOPATHIES II
Lun Yi, Su Xu, R. Soska, Anju M. Nair +9 more
2018Neuromuscular Disorders1 citations
Semantic Scholar
Histological examination of the effect of a highly phosphorylated proprietary recombinant human acid alpha-glucosidase on glycogen reduction in disease-relevant muscles of Pompe mice
Lun Yi, Su Xu, R. Gotschall, A. Schilling +8 more
20151 citations
Semantic Scholar
Exploring the use of a co-formulated pharmacological chaperone AT2220 with recombinant human acid alpha-glucosidase for Pompe disease
Richie Khanna, Su Xu, Lun Yi, R. Soska +6 more
20131 citations
Semantic Scholar
P.17.8 The co-formulation of pharmacological chaperone AT2220 with recombinant human acid alpha-glucosidase improves enzyme uptake and glycogen reduction in a mouse model of Pompe disease
Richie Khanna, Su Xu, Lee J. Pellegrino, Lun Yi +7 more
20131 citations
Semantic Scholar
Abstract P245: Synergistic effect of combination of pemigatinib with enfortumab vedotin (EV) in human bladder cancer models
Rod Hess, L. Truong, A. Chadderton, M. Frascella +2 more
2021Poster Presentations - Proffered Abstracts0 citations
Semantic Scholar
A major advance in the search for more effective therapy for Pompe disease
N. Raben, E. Ralston, Lun Yi, A. Ponery +7 more
20180 citations
Semantic Scholar
A next-generation Fabry enzyme replacement therapy: A proprietary human α-galactosidase A co-formulated with a pharmacological chaperone, AT1001, shows greater substrate reduction than standard of care in Fabry mice
Su Xu, A. Schilling, Nestor Gomez, M. Frascella +12 more
20180 citations
Semantic Scholar
A novel recombinant human acid alpha-glucosidase, ATB200, leads to greater substrate reduction and improvement in Pompe disease-relevant markers compared to alglucosidase alfa in GAA KO mice
Lun Yi, Su Xu, R. Soska, Anju M. Nair +6 more
20170 citations
Semantic Scholar
Stabilized next-generation recombinant human acid alpha-glucosidase ATB200 clears accumulated glycogen and reverses cellular dysfunction to increase functional muscle strength in a mouse model of Pompe disease
Do Hung, Lun Yi, Su Xu, R. Soska +9 more
20170 citations
Semantic Scholar
Co-administration of the pharmacological chaperone AT2221 with a proprietary recombinant human acid alfa-glucosidase leads to greater plasma exposure and substrate reduction compared to alglucosidase alfa
Richie Khanna, Su Xu, D. Hilliard, Lun Yi +13 more
20160 citations
Semantic Scholar
Subcutaneous administration of recombinant human acid α-glucosidase co-formulated with the pharmacological chaperone AT2220 leads to lysosomal uptake of rhGAA and glycogen reduction in disease-relevant tissues of mice with Pompe disease
Lun Yi, Lee J. Pellegrino, Su Xu, Jessie Feng +7 more
20140 citations
Semantic Scholar
T.P.44 The pharmacological chaperone AT2220 increases the stability of recombinant human acid α-glucosidase and leads to greater tissue uptake and glycogen reduction in a mouse model of Pompe disease
K. Valenzano, Jessie Feng, R. Soska, Lee J. Pellegrino +6 more
20120 citations
Semantic Scholar
Improved pharmacological chaperones for the treatment of neuronopathic Gaucher and Parkinson's disease
S. Clark, Gary Lee, R. Boyd, S. Sullivan +9 more
20110 citations
Semantic Scholar
Pharmacological chaperones aren't just for mutant enzymes anymore: Co-administration of AT1001 Stabilizes rh∂-Gal A, leading to greater uptake and substrate reduction in fabry patient-derived cells and GLA knockout mice
Richie Khanna, Elfrida R. Benjamin, J. Flanagan, Lee J. Pellegrino +8 more
20110 citations
Semantic Scholar
29. Genetic and pharmacological chaperone modulation of brain GCase activity affects synuclein accumulation in mice
S. Clark, Ying Sun, You-hai Xu, Lee J. Pellegrino +7 more
20100 citations
Semantic Scholar
CHEMO-RESISTANT SOFT-TISSUE SARCOMAS (STS). Experience of the Italian Cooperative Study (ICS)
G. Cecchetto, P. Grotto, M. Carli, F. Siracusa +3 more
19930 citations
Semantic Scholar

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Richie Khanna×23
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Richie Khanna — researcher profile on SciRah
K. Valenzano×23
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Lun Yi×20
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Lun Yi — researcher profile on SciRah
R. Soska×18
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Anadina Garcia×14

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Su Xu — researcher profile on SciRah

Lee J. Pellegrino×11

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D. Lockhart×11

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Do Hung — researcher profile on SciRah

A. Schilling×9

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A. Schilling — researcher profile on SciRah

R. Gotschall×9

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R. Gotschall — researcher profile on SciRah