Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Authors: Mendell, J. R.

Source: New England journal of medicine. 377(18):1713-1722

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN613444000&indx=1&recIds=ETOCRN613444000

Modeling functional decline over time in sporadic inclusion body myositis

Authors: Alfano, L. N., Yin, H., Dvorchik, I., Maus, E. G., Flanigan, K. M., Mendell, J. R., Lowes, L. P.

Source: MUSCLE AND NERVE. 55(4):526-531

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P.134a Phase 1/2a trial of delandistrogene moxeparvovec in patients with DMD: 4-year update

Authors: Mendell, J., Sahenk, Z., Lehman, K., Lowes, L., Reash, N., Iammarino, M., Alfano, L., Lewis, S., Church, K., Shell, R., Potter, R., Griffin, D., Pozsgai, E., Hogan, M., Hu, L., Mason, S., Darton, E., Rodino-Klapac, L.

Source: NEUROMUSCULAR DISORDERS. 32(S1):S102-S103

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100173057887.0x000001&indx=1&recIds=ETOCvdc_100173057887.0x000001

FP.34 Clinical outcome study of dysferlinopathy: correlation between MRI fat fraction in lower limbs and clinical outcome assessments over a 3-year period

Authors: Smith, F., Reyngoudt, H., Manera, J. Diaz, James, M., Wilson, I., de Almeida Araujo, E. Caldas, Diaz, C. Bolano, Dressman, H. Gordish, Rufibach, L., Mayhew, A., Jones, K., Campana, E. Salort, Walter, M., Stojkovic, T., Yoshimura, M., Pegoraro, E., Mendell, J., Straub, V., Blamire, A., Carlier, P.

Source: NEUROMUSCULAR DISORDERS. 32(S1):S112-S113

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100173057458.0x000001&indx=1&recIds=ETOCvdc_100173057458.0x000001

Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy

Authors: Griggs, R. C., Miller, J. P., Greenberg, C. R., Fehlings, D. L., Pestronk, A., Mendell, J. R., Moxley, R. T., King, W., Kissel, J. T., Cwik, V.

Source: Neurology. 87(20):2123-2131

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN608990729&indx=1&recIds=ETOCRN608990729

Edoxaban drug-drug interactions with ketoconazole, erythromycin, and cyclosporine

Authors: Parasrampuria, D. A., Mendell, J., Shi, M., Matsushima, N., Zahir, H., Truitt, K.

Source: BRITISH JOURNAL OF CLINICAL PHARMACOLOGY. 82(6):1591-1600

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'The Certified Duchenne Care Center Program'

Authors: Kinnett, K., Dowling, J. J., Mendell, J. R.

Source: NEUROMUSCULAR DISORDERS. 26(12):853-859

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN382202919&indx=1&recIds=ETOCRN382202919

Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up

Authors: Connolly, A. M., Florence, J. M., Zaidman, C. M., Golumbek, P. T., Mendell, J. R., Flanigan, K. M., Karachunski, P. I., Day, J. W., McDonald, C. M., Darras, B. T.

Source: MUSCLE AND NERVE. 54(4):681-689

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Clinical Follow-Up for Duchenne Muscular Dystrophy Newborn Screening: A Proposal

Authors: Kwon, J. M., Abdel-Hamid, H. Z., Al-Zaidy, S. A., Mendell, J. R., Kennedy, A., Kinnett, K., Cwik, V. A., Street, N., Bolen, J., Day, J. W.

Source: MUSCLE AND NERVE. 54(2):186-191

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN379473603&indx=1&recIds=ETOCRN379473603

Population pharmacokinetic analysis of patritumab, a HER3 inhibitor, in subjects with advanced non-small cell lung cancer (NSCLC) or solid tumors

Authors: Yoshiba, S., Jansen, M., Matsushima, N., Chen, S., Mendell, J.

Source: CANCER CHEMOTHERAPY AND PHARMACOLOGY. 77(5):987-996

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN377095801&indx=1&recIds=ETOCRN377095801

A thorough QTc study demonstrates that olmesartan medoxomil does not prolong the QTc interval

Authors: Mendell, J., Matsushima, N., O'Reilly, T. E., Lee, J.

Source: JOURNAL OF CLINICAL PHARMACOLOGY. 56(4):484-491

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN376364720&indx=1&recIds=ETOCRN376364720

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Authors: Mendell, J. R., Goemans, N., Lowes, L. P., Alfano, L. N., Berry, K., Shao, J., Kaye, E. M., Mercuri, E.

Source: ANNALS OF NEUROLOGY. 79(2):257-271

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Linear mixed-effects model of QTc prolongation for olmesartan medoxomil

Authors: Song, S., Matsushima, N., Lee, J., Mendell, J.

Source: JOURNAL OF CLINICAL PHARMACOLOGY. 56(1):96-100

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN374076977&indx=1&recIds=ETOCRN374076977

Noncoding RNA NORAD Regulates Genomic Stability by Sequestering PUMILIO Proteins

Authors: Lee, S., Kopp, F., Chang, T. C., Sataluri, A., Chen, B., Sivakumar, S., Yu, H., Xie, Y., Mendell, J. T.

Source: CELL -CAMBRIDGE MA-. 164(1):69-80

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN373976876&indx=1&recIds=ETOCRN373976876

AAV1.NT-3 gene therapy attenuates spontaneous autoimmune peripheral polyneuropathy

Authors: Yalvac, M. E., Arnold, W. D., Braganza, C., Chen, L., Mendell, J. R., Sahenk, Z.

Source: GENE THERAPY -BASINGSTOKE-. 23(1):95-102

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN373668241&indx=1&recIds=ETOCRN373668241

β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice

Authors: Pozsgai, E. R., Griffin, D. A., Heller, K. N., Mendell, J. R., Rodino-Klapac, L. R.

Source: GENE THERAPY -BASINGSTOKE-. 23(1):57-66

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN373668253&indx=1&recIds=ETOCRN373668253

Reliability and validity of active-seated: An outcome in dystrophinopathy

Authors: Lowes, L. P., Alfano, L. N., Crawfis, R., Berry, K., Yin, H., Dvorchik, I., Flanigan, K. M., Mendell, J. R.

Source: MUSCLE AND NERVE. 52(3):356-362

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN381768660&indx=1&recIds=ETOCRN381768660

An open-label, phase 1 study to evaluate the effects of hepatic impairment on edoxaban pharmacokinetics and pharmacodynamics

Authors: Mendell, J., Johnson, L., Chen, S.

Source: JOURNAL OF CLINICAL PHARMACOLOGY. 55(12):1395-1405

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN374885855&indx=1&recIds=ETOCRN374885855

Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45

Authors: Findlay, A. R., Wein, N., Kaminoh, Y., Taylor, L. E., Dunn, D. M., Mendell, J. R., King, W. M., Pestronk, A., Florence, J. M., Mathews, K. D.

Source: ANNALS OF NEUROLOGY. 77(4):668-674

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN369492300&indx=1&recIds=ETOCRN369492300

Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

Authors: Connolly, A. M., Malkus, E. C., Mendell, J. R., Flanigan, K. M., Miller, J. P., Schierbecker, J. R., Siener, C. A., Golumbek, P. T., Zaidman, C. M., Mcdonald, C. M.

Source: MUSCLE AND NERVE. 51(4):522-532

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN368994441&indx=1&recIds=ETOCRN368994441