Michael Lawlor, MD PhD

Affiliations: 
Childrens Hospital Boston, Harvard Medical School, Boston, MA, United States 
Website:
http://www.linkedin.com/pub/mike-lawlor/a/4a3/a25
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"Michael Lawlor"
Mean distance: 17.42 (cluster 24)
 
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Publications

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Karimi E, van der Borgh M, Lindqvist J, et al. (2023) Characterization of mutations in patients reveals novel nemaline myopathy disease mechanisms and omecamtiv mecarbil force effects. Biorxiv : the Preprint Server For Biology
Lawlor MW, Schoser B, Margeta M, et al. (2023) 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. Ebiomedicine. 99: 104894
Shieh PB, Kuntz NL, Dowling JJ, et al. (2023) Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial. The Lancet. Neurology. 22: 1125-1139
Slick RA, Tinklenberg JA, Sutton J, et al. (2023) Aberrations in Energetic Metabolism and Stress-Related Pathways Contribute to Pathophysiology in the Neb Conditional Knockout Mouse Model of Nemaline Myopathy. The American Journal of Pathology
Tinklenberg JA, Slick RA, Sutton J, et al. (2023) Different Mouse Models of Nemaline Myopathy Harboring Acta1 Mutations Display Differing Abnormalities Related to Mitochondrial Biology. The American Journal of Pathology
Gartz M, Haberman M, Sutton J, et al. (2023) ACTA1 H40Y mutant iPSC-derived skeletal myocytes display mitochondrial defects in an in vitro model of nemaline myopathy. Experimental Cell Research. 424: 113507
Birch SM, Lawlor MW, Conlon TJ, et al. (2023) Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy. Science Translational Medicine. 15: eabo1815
Boehler JF, Brown KJ, Beatka M, et al. (2022) Clinical potential of microdystrophin as a surrogate endpoint. Neuromuscular Disorders : Nmd
Dowling JJ, Müller-Felber W, Smith BK, et al. (2022) INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy. Journal of Neuromuscular Diseases
Gartz M, Haberman M, Prom MJ, et al. (2022) A Long-Term Study Evaluating the Effects of Nicorandil Treatment on Duchenne Muscular Dystrophy-Associated Cardiomyopathy in Mice. Journal of Cardiovascular Pharmacology and Therapeutics. 27: 10742484221088655
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