Nirmatrelvir-Ritonavir and Symptoms in Adults With Postacute Sequelae of SARS-CoV-2 Infection: The STOP-PASC Randomized Clinical Trial
Geng, Linda N, Bonilla, Hector, Hedlin, Haley, Jacobson, Karen B, Tian, Lu, Jagannathan, Prasanna, Yang, Phillip C, Subramanian, Aruna K, Liang, Jane W, Shen, Sa, Deng, Yaowei, Shaw, Blake J, Botzheim, Bren, Desai, Manisha, Pathak, Divya, Jazayeri, Yasmin, Thai, Daniel, O'Donnell, Andrew, Mohaptra, Sukanya, Leang, Zenita, Reynolds, Gabriella Z M, Brooks, Erin F, Bhatt, Ami S, Shafer, Robert W, Miglis, Mitchell G, Quach, Tom, Tiwari, Anushri, Banerjee, Anindita, Lopez, Rene N, De Jesus, Magdia, Charnas, Lawrence R, Utz, Paul J, Singh, Upinder
Published in JAMA internal medicine (01.09.2024)
Published in JAMA internal medicine (01.09.2024)
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Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy
Sherlock, Sarah P., Palmer, Jeffrey, Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Tian, Cuixia, Mah, Jean K., Muntoni, Francesco, Guglieri, Michela, Butterfield, Russell J., Charnas, Lawrence, Marraffino, Shannon
Published in Scientific reports (05.11.2022)
Published in Scientific reports (05.11.2022)
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Mutations of CASK cause an X-linked brain malformation phenotype with microcephaly and hypoplasia of the brainstem and cerebellum
Chizhikov, Victor V, Uyanik, Gökhan, Kuechler, Alma, Kutsche, Kerstin, Flubacher, Armin, Charnas, Lawrence R, Golden, Jeffrey A, Haas, Carola A, Frank, Ulrich, Ullmann, Reinhard, Klopocki, Eva, Dobyns, William B, Sudi, Jyotsna, Wimplinger, Isabella, Christian, Susan L, Horn, Denise, Najm, Juliane
Published in Nature genetics (01.09.2008)
Published in Nature genetics (01.09.2008)
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Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy
Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Mah, Jean K., Campbell, Craig, Guglieri, Michela, Muntoni, Francesco, Takeshima, Yasuhiro, McDonald, Craig M., Kostera-Pruszczyk, Anna, Karachunski, Peter, Butterfield, Russell J., Mercuri, Eugenio, Fiorillo, Chiara, Bertini, Enrico S., Tian, Cuixia, Statland, Jeffery, Sadosky, Alesia B., Purohit, Vivek S., Sherlock, Sarah P., Palmer, Jeffrey P., Binks, Michael, Charnas, Lawrence, Marraffino, Shannon, Wong, Brenda L.
Published in Neuromuscular disorders : NMD (01.06.2020)
Published in Neuromuscular disorders : NMD (01.06.2020)
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Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial
Muntoni, Francesco, Guglieri, Michela, Mah, Jean K., Wagner, Kathryn R., Brandsema, John F., Butterfield, Russell J., McDonald, Craig M., Mayhew, Anna G., Palmer, Jeffrey P., Marraffino, Shannon, Charnas, Lawrence, Mercuri, Eugenio
Published in PloS one (23.08.2022)
Published in PloS one (23.08.2022)
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Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999
Peters, Charles, Charnas, Lawrence R., Tan, Ye, Ziegler, Richard S., Shapiro, Elsa G., DeFor, Todd, Grewal, Satkiran S., Orchard, Paul J., Abel, Susan L., Goldman, Anne I., Ramsay, Norma K.C., Dusenbery, Kathryn E., Loes, Daniel J., Lockman, Lawrence A., Kato, Shunichi, Aubourg, Patrick R., Moser, Hugo W., Krivit, William
Published in Blood (01.08.2004)
Published in Blood (01.08.2004)
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Current state of cardiac troponin testing in Duchenne muscular dystrophy cardiomyopathy: review and recommendations from the Parent Project Muscular Dystrophy expert panel
Spurney, Christopher F, Ascheim, Deborah, Charnas, Lawrence, Cripe, Linda, Hor, Kan, King, Nicholas, Kinnett, Kathi, McNally, Elizabeth M, Sauer, John-Michael, Sweeney, Lee, Villa, Chet, Markham, Larry W
Published in Open heart (24.03.2021)
Published in Open heart (24.03.2021)
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Hematopoietic Cell Therapy for Metabolic Disease
Orchard, Paul J., MD, Blazar, Bruce R., MD, Wagner, John, MD, Charnas, Lawrence, MD, PhD, Krivit, William, MD, PhD, Tolar, Jakub, MD, PhD
Published in The Journal of pediatrics (01.10.2007)
Published in The Journal of pediatrics (01.10.2007)
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Progressive brainstem compression in an infant with neurocutaneous melanosis and Dandy-Walker complex following ventriculoperitoneal shunt placement for hydrocephalus. Case report
McClelland, 3rd, Shearwood, Charnas, Lawrence R, SantaCruz, Karen S, Garner, Hart P, Lam, Cornelius H
Published in Journal of neurosurgery (01.12.2007)
Published in Journal of neurosurgery (01.12.2007)
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Jackson-Weiss and Crouzon syndromes are allelic with mutations in fibroblast growth factor receptor 2
Jabs, Ethylin Wang, Li, Xiang, Scott, Alan F, Meyers, Gregory, Chen, Wendy, Eccles, Michael, Mao, Jen-i, Charnas, Lawrence R, Jackson, Charles E, Jaye, Michael
Published in Nature genetics (01.11.1994)
Published in Nature genetics (01.11.1994)
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Severe methylenetetrahydrofolate reductase (MTHFR) deficiency: a case report of nonclassical homocystinuria
Bishop, Lisa, Kanoff, Richard, Charnas, Lawrence, Krenzel, Charles, Berry, Susan A, Schimmenti, Lisa A
Published in Journal of child neurology (01.07.2008)
Published in Journal of child neurology (01.07.2008)
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Physical mapping and genomic structure of the Lowe syndrome gene OCRL1
NUSSBAUM, R. L, ORRISON, B. M, JÄNNE, P. A, CHARNAS, L, CHINAULT, A. C
Published in Human genetics (01.02.1997)
Published in Human genetics (01.02.1997)
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Novel approaches to analysis of the North Star Ambulatory Assessment : Observations from a phase 2 trial
Muntoni, Francesco, Guglieri, Michela, Mah, Jean K, Wagner, Kathryn R, Brandsema, John F, Butterfield, Russell J, McDonald, Craig M, Mayhew, Anna G, Palmer, Jeffrey P, Marraffino, Shannon, Charnas, Lawrence, Mercuri, Eugenio
Published in PloS one (23.08.2022)
Published in PloS one (23.08.2022)
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Journal Article
Novel approaches to analysis of the North Star Ambulatory Assessment
Muntoni, Francesco, Guglieri, Michela, Mah, Jean K, Wagner, Kathryn R, Brandsema, John F, Butterfield, Russell J, McDonald, Craig M, Mayhew, Anna G, Palmer, Jeffrey P, Marraffino, Shannon, Charnas, Lawrence, Mercuri, Eugenio
Published in PloS one (23.08.2022)
Published in PloS one (23.08.2022)
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Journal Article
Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab
Sherlock, Sarah P., Palmer, Jeffrey, Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Bertini, Enrico, Tian, Cuixia, Mah, Jean K., Kostera-Pruszczyk, Anna, Muntoni, Francesco, Guglieri, Michela, Brandsema, John F., Mercuri, Eugenio, Butterfield, Russell J., McDonald, Craig M., Charnas, Lawrence, Marraffino, Shannon
Published in Journal of neurology (01.08.2022)
Published in Journal of neurology (01.08.2022)
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The PAX LC Trial: A Decentralized, Phase 2, Randomized, Double-Blind Study of Nirmatrelvir/Ritonavir Compared with Placebo/Ritonavir for Long COVID
Krumholz, Harlan M., Sawano, Mitsuaki, Bhattacharjee, Bornali, Caraballo, César, Khera, Rohan, Li, Shu-Xia, Herrin, Jeph, Coppi, Andreas, Holub, Julie, Henriquez, Yashira, Johnson, Maria A., Goddard, Theresa B., Rocco, Erica, Hummel, Amy C., AL Mouslmani, Mohammad, Putrino, David F., Carr, Kevin D., Carvajal-Gonzalez, Santos, Charnas, Lawrence, De Jesus, Magdia, Ziegler, Frank W., Iwasaki, Akiko
Published in The American journal of medicine (10.05.2024)
Published in The American journal of medicine (10.05.2024)
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Intrathecal Gene Therapy for Giant Axonal Neuropathy
Bharucha-Goebel, Diana X., Todd, Joshua J., Saade, Dimah, Norato, Gina, Jain, Minal, Lehky, Tanya, Bailey, Rachel M., Chichester, Jessica A., Calcedo, Roberto, Armao, Diane, Foley, A. Reghan, Mohassel, Payam, Tesfaye, Eshetu, Carlin, Bradley P., Seremula, Beth, Waite, Melissa, Zein, Wadih M., Huryn, Laryssa A., Crawford, Thomas O., Sumner, Charlotte J., Hoke, Ahmet, Heiss, John D., Charnas, Lawrence, Hooper, Jody E., Bouldin, Thomas W., Kang, Elizabeth M., Rybin, Denis, Gray, Steven J., Bönnemann, Carsten G.
Published in The New England journal of medicine (21.03.2024)
Published in The New England journal of medicine (21.03.2024)
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