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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
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, Mouslmani MA, 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
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.
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.
Corrigendum to "Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy" [Neuromuscular Disorders, Vol. 30 (6) 2020, 492-502].
Wagner KR, Abdel-Hamid HZ, Mah JK, Campbell C, Guglieri M, Muntoni F, Takeshima Y, McDonald CM, Kostera-Pruszczyk A, Karachunski P, Butterfield RJ, Mercuri E, Fiorillo C, Bertini ES, Tian C, Statland J, Sadosky AB, Purohit VS, Sherlock SP, Palmer JP, Binks M, Charnas L, Marraffino S, Wong BL. Wagner KR, et al. Among authors: charnas l. Neuromuscul Disord. 2021 Feb;31(2):167-168. doi: 10.1016/j.nmd.2021.01.001. Epub 2021 Jan 13. Neuromuscul Disord. 2021. PMID: 33451933 No abstract available.
Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy.
Wagner KR, Abdel-Hamid HZ, Mah JK, Campbell C, Guglieri M, Muntoni F, Takeshima Y, McDonald CM, Kostera-Pruszczyk A, Karachunski P, Butterfield RJ, Mercuri E, Fiorillo C, Bertini ES, Tian C, Statland J, Sadosky AB, Purohit VS, Sherlock SP, Palmer JP, Binks M, Charnas L, Marraffino S, Wong BL. Wagner KR, et al. Among authors: charnas l. Neuromuscul Disord. 2020 Jun;30(6):492-502. doi: 10.1016/j.nmd.2020.05.002. Epub 2020 May 19. Neuromuscul Disord. 2020. PMID: 32522498 Free article. Clinical Trial.
58 results