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Page 1
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: miller w. 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.
Intrahepatic Cholestasis Is a Clinically Significant Feature Associated with Natural History of X-Linked Myotubular Myopathy (XLMTM): A Case Series and Biopsy Report.
Molera C, Sarishvili T, Nascimento A, Rtskhiladze I, Muñoz Bartolo G, Fernández Cebrián S, Valverde Fernández J, Muñoz Cabello B, Graham RJ, Miller W, Sepulveda B, Kamath BM, Meng H, Lawlor MW. Molera C, et al. Among authors: miller w. J Neuromuscul Dis. 2022;9(1):73-82. doi: 10.3233/JND-210712. J Neuromuscul Dis. 2022. PMID: 34366366 Free PMC article.
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: miller w. J Neuromuscul Dis. 2022;9(4):503-516. doi: 10.3233/JND-210781. J Neuromuscul Dis. 2022. PMID: 35694931 Free PMC article.
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
Lawlor MW, Schoser B, Margeta M, Sewry CA, Jones KA, Shieh PB, Kuntz NL, Smith BK, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Blaschek A, Neuhaus S, Foley AR, Saade DN, Tsuchiya E, Qasim UR, Beatka M, Prom MJ, Ott E, Danielson S, Krakau P, Kumar SN, Meng H, Vanden Avond M, Wells C, Gordish-Dressman H, Beggs AH, Christensen S, Conner E, James ES, Lee J, Sadhu C, Miller W, Sepulveda B, Varfaj F, Prasad S, Rico S. Lawlor MW, et al. Among authors: miller w. EBioMedicine. 2024 Jan;99:104894. doi: 10.1016/j.ebiom.2023.104894. Epub 2023 Dec 12. EBioMedicine. 2024. PMID: 38086156 Free PMC article.
Correction to: CXCR2 antagonist navarixin in combination with pembrolizumab in select advanced solid tumors: a phase 2 randomized trial.
Armstrong AJ, Geva R, Chung HC, Lemech C, Miller WH Jr, Hansen AR, Lee JS, Tsai F, Solomon BJ, Kim TM, Rolfo C, Giranda V, Ren Y, Liu F, Kandala B, Freshwater T, Wang JS. Armstrong AJ, et al. Among authors: miller wh jr. Invest New Drugs. 2024 May 20. doi: 10.1007/s10637-024-01444-0. Online ahead of print. Invest New Drugs. 2024. PMID: 38767685 No abstract available.
Teratogenesis, Perinatal, and Neurodevelopmental Outcomes After In Utero Exposure to Antiseizure Medication: Practice Guideline From the AAN, AES, and SMFM.
Pack AM, Oskoui M, Williams Roberson S, Donley DK, French J, Gerard EE, Gloss D, Miller WR, Munger Clary HM, Osmundson SS, McFadden B, Parratt K, Pennell PB, Saade G, Smith DB, Sullivan K, Thomas SV, Tomson T, Dolan O'Brien M, Botchway-Doe K, Silsbee HM, Keezer MR. Pack AM, et al. Among authors: miller wr. Neurology. 2024 Jun;102(11):e209279. doi: 10.1212/WNL.0000000000209279. Epub 2024 May 15. Neurology. 2024. PMID: 38748979
Alternative iron-depleted media for cefiderocol susceptibility testing.
Eales BM, Smith JE, Pouya N, Teran NS, Miller WR, Tam VH. Eales BM, et al. Among authors: miller wr. Int J Antimicrob Agents. 2024 May 7:107193. doi: 10.1016/j.ijantimicag.2024.107193. Online ahead of print. Int J Antimicrob Agents. 2024. PMID: 38723696 No abstract available.
Creation and characterization of novel rat model for recessive dystrophic epidermolysis bullosa: Frameshift mutation of the Col7a1 gene leads to severe blistered phenotype.
Stone W, Strege C, Miller W, Geurts AM, Grzybowski M, Riddle M, Lees C, Eide C, Keene DR, Tufa SF, Seelig D, McGrath J, Tolar J. Stone W, et al. Among authors: miller w. PLoS One. 2024 May 9;19(5):e0302991. doi: 10.1371/journal.pone.0302991. eCollection 2024. PLoS One. 2024. PMID: 38722855 Free PMC article.
6,081 results