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Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Shieh PB, Kuntz NL, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Servais L, Smith BK, Muntoni F, Blaschek A, Foley AR, Saade DN, Neuhaus S, Alfano LN, Beggs AH, Buj-Bello A, Childers MK, Duong T, Graham RJ, Jain M, Coats J, MacBean V, James ES, Lee J, Mavilio F, Miller W, Varfaj F, Murtagh M, Han C, Noursalehi M, Lawlor MW, Prasad S, Rico S. Shieh PB, et al. Among authors: noursalehi m. Lancet Neurol. 2023 Dec;22(12):1125-1139. doi: 10.1016/S1474-4422(23)00313-7. Lancet Neurol. 2023. PMID: 37977713 Free article. Clinical Trial.
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
Dowling JJ, Müller-Felber W, Smith BK, Bönnemann CG, Kuntz NL, Muntoni F, Servais L, Alfano LN, Beggs AH, Bilder DA, Blaschek A, Duong T, Graham RJ, Jain M, Lawlor MW, Lee J, Coats J, Lilien C, Lowes LP, MacBean V, Neuhaus S, Noursalehi M, Pitts T, Finlay C, Christensen S, Rafferty G, Seferian AM, Tsuchiya E, James ES, Miller W, Sepulveda B, Vila MC, Prasad S, Rico S, Shieh PB; INCEPTUS investigators. Dowling JJ, et al. Among authors: noursalehi m. J Neuromuscul Dis. 2022;9(4):503-516. doi: 10.3233/JND-210781. J Neuromuscul Dis. 2022. PMID: 35694931 Free PMC article.
Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis.
Graham RJ, Muntoni F, Hughes I, Yum SW, Kuntz NL, Yang ML, Byrne BJ, Prasad S, Alvarez R, Genetti CA, Haselkorn T, James ES, LaRusso LB, Noursalehi M, Rico S, Beggs AH. Graham RJ, et al. Among authors: noursalehi m. Arch Dis Child. 2020 Apr;105(4):332-338. doi: 10.1136/archdischild-2019-317910. Epub 2019 Sep 4. Arch Dis Child. 2020. PMID: 31484632 Free PMC article.
Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance.
Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Duong T, et al. Among authors: noursalehi m. J Neuromuscul Dis. 2021;8(1):63-77. doi: 10.3233/JND-200479. J Neuromuscul Dis. 2021. PMID: 32925083 Free PMC article.
Effect of enteral feeding with eicosapentaenoic acid, gamma-linolenic acid, and antioxidants in patients with acute respiratory distress syndrome. Enteral Nutrition in ARDS Study Group.
Gadek JE, DeMichele SJ, Karlstad MD, Pacht ER, Donahoe M, Albertson TE, Van Hoozen C, Wennberg AK, Nelson JL, Noursalehi M. Gadek JE, et al. Among authors: noursalehi m. Crit Care Med. 1999 Aug;27(8):1409-20. doi: 10.1097/00003246-199908000-00001. Crit Care Med. 1999. PMID: 10470743 Clinical Trial.
Hydroxymethylation profile of cell-free DNA is a biomarker for early colorectal cancer.
Walker NJ, Rashid M, Yu S, Bignell H, Lumby CK, Livi CM, Howell K, Morley DJ, Morganella S, Barrell D, Caim S, Gosal W, Füllgrabe J, Charlesworth TJ, Vasquez L, Ahdesmäki M, Eizenga J, Prabhat P, Proutski V, Murat-Onana ML, Greenwood CJ, Kirkwood L, Maisuria-Armer M, Li M, Coats E, Winfield V, MacBean L, Stock T, Tomé-Fernandez A, Chan Y, Sheikh N, Golder P, Steward M, Ost TWB, Stewart D, Vilella A, Noursalehi M, Paten B, Lucarelli D, Mason J, Ridge G, Mellad J, Shirodkar S, Balasubaramanian S, Holbrook JD. Walker NJ, et al. Among authors: noursalehi m. Sci Rep. 2022 Oct 4;12(1):16566. doi: 10.1038/s41598-022-20975-1. Sci Rep. 2022. PMID: 36195648 Free PMC article.
Multinational, double-blind, phase III study of prednisone and either satraplatin or placebo in patients with castrate-refractory prostate cancer progressing after prior chemotherapy: the SPARC trial.
Sternberg CN, Petrylak DP, Sartor O, Witjes JA, Demkow T, Ferrero JM, Eymard JC, Falcon S, Calabrò F, James N, Bodrogi I, Harper P, Wirth M, Berry W, Petrone ME, McKearn TJ, Noursalehi M, George M, Rozencweig M. Sternberg CN, et al. Among authors: noursalehi m. J Clin Oncol. 2009 Nov 10;27(32):5431-8. doi: 10.1200/JCO.2008.20.1228. Epub 2009 Oct 5. J Clin Oncol. 2009. PMID: 19805692 Clinical Trial.
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