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Charnas, Lawrence
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1

The PAX LC Trial: A Decentralized, Phase 2, Randomized, Dou..:

Krumholz, Harlan M. ; Sawano, Mitsuaki ; Bhattacharjee, Bornali...
The American Journal of Medicine.  , 2024
Link: https://doi.org/10.1016/..
 
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2

Novel approaches to analysis of the North Star Ambulatory A..:

Muntoni, Francesco ; Guglieri, Michela ; Mah, Jean K....
PLOS ONE.  17 (2022)  8 - p. e0272858 , 2022
Link: https://doi.org/10.1371/..
 
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3

Dual-energy X-ray absorptiometry measures of lean body mass..:

Sherlock, Sarah P. ; Palmer, Jeffrey ; Wagner, Kathryn R....
Scientific Reports.  12 (2022)  1 - p. , 2022
Link: https://doi.org/10.1038/..
 
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4

Quantitative magnetic resonance imaging measures as biomark..:

Sherlock, Sarah P. ; Palmer, Jeffrey ; Wagner, Kathryn R....
Journal of Neurology.  269 (2022)  8 - p. 4421-4435 , 2022
Link: https://doi.org/10.1007/..
 
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5

Current state of cardiac troponin testing in Duchenne muscu..:

Spurney, Christopher F ; Ascheim, Deborah ; Charnas, Lawrence...
Open Heart.  8 (2021)  1 - p. e001592 , 2021
Link: https://doi.org/10.1136/..
 
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6

Safety and Disease Monitoring Biomarkers in Duchenne Muscul..:

Wagner, Kathryn R ; Guglieri, Michaela ; Ramaiah, Shashi K...
Biomarkers in Medicine.  15 (2021)  15 - p. 1389-1396 , 2021
Link: https://doi.org/10.2217/..
 
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7

Corrigendum to "Randomized phase 2 trial and open-label ext..:

Wagner, Kathryn R. ; Abdel-Hamid, Hoda Z. ; Mah, Jean K....
Neuromuscular Disorders.  31 (2021)  2 - p. 167-168 , 2021
Link: https://doi.org/10.1016/..
 
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8

Randomized phase 2 trial and open-label extension of domagr..:

Wagner, Kathryn R. ; Abdel-Hamid, Hoda Z. ; Mah, Jean K....
Neuromuscular Disorders.  30 (2020)  6 - p. 492-502 , 2020
Link: https://doi.org/10.1016/..
 
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9

How a patient advocacy group developed the first proposed d..:

Furlong, Pat ; Bridges, John F. P. ; Charnas, Lawrence...
Orphanet Journal of Rare Diseases.  10 (2015)  1 - p. , 2015
Link: https://doi.org/10.1186/..
 
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10

X-linked adrenoleukodystrophy: ABCD1 de novo mutations and ..:

Wang, Ying ; Busin, Rachel ; Reeves, Catherine...
Molecular Genetics and Metabolism.  104 (2011)  1-2 - p. 160-166 , 2011
Link: https://doi.org/10.1016/..
 
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11

Enzyme replacement therapy corrects Lysosomal Acid Lipase D..:

Grabowski, Gregory ; Du, Hong ; Pescatore, Brian...
Molecular Genetics and Metabolism.  102 (2011)  2 - p. S19 , 2011
Link: https://doi.org/10.1016/..
 
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12

26. A re-analysis of disease stage progression in Krabbe di..:

Charnas, Lawrence ; Nair, Nitin ; Mensah, Robert
Molecular Genetics and Metabolism.  99 (2010)  2 - p. S13 , 2010
Link: https://doi.org/10.1016/..
 
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13

98. Chitotriosidase as a biomarker of cerebral adrenoleukod..:

Orchard, Paul J. ; Basso, Lisa ; Kivisto, Teresa...
Molecular Genetics and Metabolism.  99 (2010)  2 - p. S28 , 2010
Link: https://doi.org/10.1016/..
 
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14

130. Neuropsychological outcomes in Hurler syndrome: 7–20 y..:

Shapiro, Elsa ; Bjoraker, Kendra ; Guler, Evren...
Molecular Genetics and Metabolism.  96 (2009)  2 - p. S40-S41 , 2009
Link: https://doi.org/10.1016/..
 
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23. Comparison of concordance ratios of symptom onset in me..:

Charnas, Lawrence ; Shapiro, Elsa
Molecular Genetics and Metabolism.  96 (2009)  2 - p. S17 , 2009
Link: https://doi.org/10.1016/..
 
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