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Year Number of Results
1977 1
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1994 4
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59 results

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Page 1
Intrathecal Gene Therapy for Giant Axonal Neuropathy.
Bharucha-Goebel DX, Todd JJ, Saade D, Norato G, Jain M, Lehky T, Bailey RM, Chichester JA, Calcedo R, Armao D, Foley AR, Mohassel P, Tesfaye E, Carlin BP, Seremula B, Waite M, Zein WM, Huryn LA, Crawford TO, Sumner CJ, Hoke A, Heiss JD, Charnas L, Hooper JE, Bouldin TW, Kang EM, Rybin D, Gray SJ, Bönnemann CG; GAN Trial Team. Bharucha-Goebel DX, et al. Among authors: charnas l. N Engl J Med. 2024 Mar 21;390(12):1092-1104. doi: 10.1056/NEJMoa2307952. N Engl J Med. 2024. PMID: 38507752
Nirmatrelvir-Ritonavir and Symptoms in Adults With Postacute Sequelae of SARS-CoV-2 Infection: The STOP-PASC Randomized Clinical Trial.
Geng LN, Bonilla H, Hedlin H, Jacobson KB, Tian L, Jagannathan P, Yang PC, Subramanian AK, Liang JW, Shen S, Deng Y, Shaw BJ, Botzheim B, Desai M, Pathak D, Jazayeri Y, Thai D, O'Donnell A, Mohaptra S, Leang Z, Reynolds GZM, Brooks EF, Bhatt AS, Shafer RW, Miglis MG, Quach T, Tiwari A, Banerjee A, Lopez RN, De Jesus M, Charnas LR, Utz PJ, Singh U. Geng LN, et al. Among authors: charnas lr. JAMA Intern Med. 2024 Sep 1;184(9):1024-1034. doi: 10.1001/jamainternmed.2024.2007. JAMA Intern Med. 2024. PMID: 38848477 Free PMC article. Clinical Trial.
Hematopoietic cell therapy for metabolic disease.
Orchard PJ, Blazar BR, Wagner J, Charnas L, Krivit W, Tolar J. Orchard PJ, et al. Among authors: charnas l. J Pediatr. 2007 Oct;151(4):340-6. doi: 10.1016/j.jpeds.2007.04.054. J Pediatr. 2007. PMID: 17889065 Review. No abstract available.
The oculocerebrorenal syndrome of Lowe.
Charnas LR, Gahl WA. Charnas LR, et al. Adv Pediatr. 1991;38:75-107. Adv Pediatr. 1991. PMID: 1927708 Review. No abstract available.
The PAX LC Trial: A Decentralized, Phase 2, Randomized, Double-Blind Study of Nirmatrelvir/Ritonavir Compared with Placebo/Ritonavir for Long COVID.
Krumholz HM, Sawano M, Bhattacharjee B, Caraballo C, Khera R, Li SX, Herrin J, Coppi A, Holub J, Henriquez Y, Johnson MA, Goddard TB, Rocco E, Hummel AC, Al Mouslmani M, Putrino DF, Carr KD, Carvajal-Gonzalez S, Charnas L, De Jesus M, Ziegler FW 3rd, Iwasaki A. Krumholz HM, et al. Among authors: charnas l. Am J Med. 2024 May 10:S0002-9343(24)00271-7. doi: 10.1016/j.amjmed.2024.04.030. Online ahead of print. Am J Med. 2024. PMID: 38735354 Free article.
Current state of cardiac troponin testing in Duchenne muscular dystrophy cardiomyopathy: review and recommendations from the Parent Project Muscular Dystrophy expert panel.
Spurney CF, Ascheim D, Charnas L, Cripe L, Hor K, King N, Kinnett K, McNally EM, Sauer JM, Sweeney L, Villa C, Markham LW. Spurney CF, et al. Among authors: charnas l. Open Heart. 2021 Mar;8(1):e001592. doi: 10.1136/openhrt-2021-001592. Open Heart. 2021. PMID: 33762424 Free PMC article. Review.
Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial.
Muntoni F, Guglieri M, Mah JK, Wagner KR, Brandsema JF, Butterfield RJ, McDonald CM, Mayhew AG, Palmer JP, Marraffino S, Charnas L, Mercuri E. Muntoni F, et al. Among authors: charnas l. PLoS One. 2022 Aug 23;17(8):e0272858. doi: 10.1371/journal.pone.0272858. eCollection 2022. PLoS One. 2022. PMID: 35998119 Free PMC article. Clinical Trial.
Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab.
Sherlock SP, Palmer J, Wagner KR, Abdel-Hamid HZ, Bertini E, Tian C, Mah JK, Kostera-Pruszczyk A, Muntoni F, Guglieri M, Brandsema JF, Mercuri E, Butterfield RJ, McDonald CM, Charnas L, Marraffino S. Sherlock SP, et al. Among authors: charnas l. J Neurol. 2022 Aug;269(8):4421-4435. doi: 10.1007/s00415-022-11084-0. Epub 2022 Apr 8. J Neurol. 2022. PMID: 35396602 Free PMC article. Clinical Trial.
59 results