Results 31 to 40 of about 4,580 (188)
Nusinersen versus sham control in infantile-onset spinal muscular atrophy [PDF]
, 2017 BACKGROUND: Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein.Stein, S., Finkel, R. S., Allen, V., Nelson, L., Gomez Garcia de la Banda, M. B., Z. John Zhong, Patnaik, S, Gidaro, T., Siener, C., O'Reardon, K., Austin, ED, Cruz, R, Kuntz, Nancy L., Yan, C. S., Endsley, J. D., Wenderickx, B., Proud, C., Martinez Bermejo, A., Ito, Y., Perez, J, Fukuda, N., Dunaway, S., Gadeken, R, Case, L., Herbert, K. J., Laine, R, Kramer, S, Sethna, N, Welsh, L., Al-Ghamdi, F, Kim, H., Finkel, RS, Chan, S., Case, L, Shun, T. N., Popolizio, M, Nagata, S., Jo, H., Michoulas, A., Carry, T., Wider, S., Richard S. Finkel, McElroy, D., Doumit, M. A., Nishikawa, A., Gilbert, G., Hen, J, Aziz-Zaman, S, Smith, E., Nelson, N, Johnson, N., DeCock, K., Staudt, L., Marra, J., Parsons, J., de Los Angeles Tormos Munoz, M., Dastgir, J., Ikai, T., Kandula, T., Bishop, K, Anand, P, Kurz, J., Eckenweiler, M., Topaloglu, H, Kelly, C., Popolizio, M., Oriyama, K., Sigurdardottir, L. Y., Al-Ghamdi, F., Golumbek, PT, ENDEAR Study Group, Carroll, K. M., Blomgren, C., Aziz-Zaman, S., Lilien, C., Quigley, J, Paulose, S., Eldblom, J., Mirek, E., Trucco, F., Choi, S. A., Miki, M., Ollievier, Q., Fuhr, P., Gunbey, C., Lord-Halvorson, L. S., Coppens, S., Benjamin, D., Engelstad, K, Kuntz, Nancy L, Russman, B, Servais, L., Golumbek, P. T., Rao, L., Ardicli, D., Wildon Farwell, Darryl C. De Vivo, Purse, B., Lee, J. S., Zaidman, C. M., Ki, N. Y., Bradley, WG, Takeshita, A., Marra, J, Connolly, Anne M, Shibano, T., Salazar, R, Tran, V., Nelson, N., Ingelmo, P. M., Rucian, A. F., Sanabria, L., Skura, C., Nicolarsen, S., O'Brien, J., Sampson, J., Zielinski, D., Cruz, R., Castro, D., Konuskan, B., Alvin, H. C. C., Eduardo Tizzano, Taicher, B, Toms, M., Benjamin, D, Arakawa, R., Lee, T., Ghosh, P, Holuba La Marca, N., Dowling, J., Coates, J, So, S., Shin, H. I., Ito, S., De Vivo, D. C., Garcia Guixot, S., De Sanctis, R., Van Coster, R., Berde, C, Eto, K., Basil T. Darras, Mercuri, E, D'Souza, G., Yiu, E. M., Schneider, E., Fujino, T., Munell Casadesus, F., Frank, A., Johnson, H., Stella, C., Teoh, H. L., Chou, Y. H., Proud, C, Saito, K, Sivo, S., Yamauchi, A., Vens, D., Chae, J. H., Chung, A. C., Kasper, M., Wand, L, Bishop, Kathie, Farrar, Michelle ; https://orcid.org/, Parziale, N., Villano, D., Rambaud, J., DiBella, D., Janice, I. J. K., Shieh, P., Baily, S., Bishop, K., Tulinius, Mar, Welsh, L, Shea, S., Lanteri, P., Kauk, M., Servais, L, Roberts, C., Wood, C., Sakamuri, S, Leon, W., Shih, H. H., Sanjanwala, B., Matsumaru, S., DeArmey, S., Pena, L. D. M., Ordonez, G., Dastgir, J, Milleson, C., Gargaun, E., Weimer, L, Bennett, C. F., Kaneko, H., Connolly, AM, del Pilar Tirado Requero, M., Glanzman, A. M., Yvonne, Y., Topaloglu, H., De Valle, K. L., Epstein, L., Gratacos Vinola, M., Yildiz Sarikaya, F. G., Coates, J., Flickinger, J., Schneider, Eugene, ENDEAR Study Group, the, Voit, T., Khandji, A, Planas Pascual, B., Berry, D., Scoto, M., Zigmont, J., Shimomura, H., Wootton, J, De Vivo, Dominique, Weber-Guzman, F., Dorban, F., Manzur, A., Chen, T. H., Harrington, T., Gormley, M., Berde, C., Bennett, C Frank, Zaidman, CM, Srour, M., Yang, M., Sethna, N., Taicher, B., del Mar Melendez Plumed, M., C. Frank Bennett, Vanlander, Arnaud, Sampson, J, De Vivo, DC, Kandula, Tejaswi ; https://orcid.org/, Garaventa, A., Salazar, R., Tesi Rocha, A. C., Selby, V., Temucin, C. M., Bielsky, A., Constantinescu, A, Matsushima, N., Roberts, C, Szelag, H., Rupprich, K., Ortiz-Miller, C, Bodensteriner, J. B., Liang, W. C., Chiriboga, CA, Mayer, M., Saito, Kayoko, Choi, H. W., Montes, J., Topaloglu, Haluk, Chiba, Y., Graham, R., Turner, J., Chung, H. W., Ju, Y. H., Pechmann, A., Cappell, J, Perez, J., Krüger, M., Gilabert, S., Tulinius, M., De Vos voor, E., Michael, E., Berthon-Jones, N., Farwell, Wildon, Engelstad, K., Lim, B. C., Kuntz, N. L., Wand, L., Arpin, S., Harrington, T, Connelly, AM, Graham, R, Aguilar, C., Homi, M. M., Booker, K., Fiorillo, C., Christiaens, A., Luigetti, M., Finanger, E, Zeng, Y. S., Chiriboga, Claudia A., Kitsuwa-Lowe, J., Pedermonte, M., Murphy, A., Finanger, E., Haluk Topaloglu, Tulinius, M, Muntoni, F., McFall, D, D'Souza, G, Hirasawa, K., Takeshima, Y., Lanzillotta, V., Schroers, E. S., Civitello, M., Anne M. Connolly, Gallardo, M., Sarah Gheuens, Sanjanwala, B, Sugimoto, K., Allan M. Glanzman, King, C., Tanyildiz, M., Zhong, ZJ, Van Der Looven, R., Gee, R, Tizzano, Eduardo, Connolly, Anne M., Eugene Schneider, Montes, J, Darin, N., Piastra Marco, Said, J. W., Purse, B, Schneider, E, Zhong, Z. J., Dunaway, S, Ryan, M., Otani, Y., Gormley, M, Day, JW, Brandsema, J., Toro Tamargo, E., Cowie, M., Piastra, M., Mayne, K., Selby, K., Kroksmark, A. K., Bennett, CF, Day, J. W., Pena, LDM, Shell, R., Zuffi, A., De Vivo, D. C., Iannaccone, S. T., Herbert, A., Ordonez, G, Rao, V., Yilmaz, O. T., Chun, S. M., Roland, E., Koelbel, H., del Mar Garcia Romero, M., Gibbons, M., Bishop, Kathie M., Lofra, R. M., Vogt, S., Ricotti, V., Kitsuwa-Lowe, J, Zhong, Z John, Joseph, C. K. W., Homi, MM, Trest, S., Lister, PH., Karaduman, A. A., Finkel, Richard S., Siener, C, Morrison, M., Rivera, G., Krueger, J., Manzitti, C., Haliloglu, V. G., Davis, C. S., Kayoko Saito, Zinn, M., Pasternak, A., Martinez Moreno, M. M., Schara, U., Patnaik, S., Alavarez Molinero, M., Liew, W, Rooze, S., Vajsar, J., Tanaka, Y., Connie, W. Y., Johnson, C., Chou, P. C., Camuto, A., De Vivo, Darryl C., Schroth, MK, Spain, T., Wootton, J., Fanelli, L., Léger, P. L., Duong, TT, Sato, T., Son, W. S., Quigley, J., Ortiz-Miller, C., Kirschner, J, Holuba La Marca, N, Ramsey, D., Andres, B., Kichula, E., Szelag, H, Kirschner, J., Deconinck, N., Rausch, N., Wendy, L. W. M., Bertoli, M., Russman, B., Glanzman, Allan M., Verhelst, H., Zimmerman, C., Woodcock, I., Liew, W., Souris, M., Eugenio Mercuri, Sofou, K., Turgeon-Desilet, S., Tacchetti, P., Glanzman, Allan M, Straub, V., Paulose, S, McFall, D., Ross, A, Chiriboga, C. A., Gadeken, R., Mirek, E, Smith, L., Yanagishita, T., Adang, L., Sakamuri, S., Ferreira Sampaio, H. A., Farrow-Gillespie, A., Anand, P., Rippberger, B., Richardson, S., Tennekoon, G., Weimer, L., Tachikawa, T., VanderVeen, G., Shu, F., Hsu, J. H., Poulin, C., Ng, C. S., Gheuens, S., Newcomb, T. M., Chui-San, M. N., Scuplak, S. M., Bradley, W. G., Oskoui, M., Gheuens, Sarah, Virginia, W. C., Gheuens, S, Kimber, E., Janbernd Kirschner, Farwell, W, Verhelst, Helene, Hen, J., Della Marina, A., Kirschner, Janbernd, Kim, S. Y., Saito, K., Wang, H. Y., Goldman, S., Yildirim, M., Kathie Bishop, Frank, A, Tesi Rocha, AC, Smith, E, Shell, R, Claudia A. Chiriboga, Bruno, C., Milleson, C, Bennett, C. Frank, Laine, R., Darras, Basil T, Van Coster, Rudy, Vialle, R., Mayhew, A. G., Farwell, W., Serdaroglu, E., Ghosh, P., Cook, D., Pisco Domingos, J., Ganfuss, A., Constantinescu, A., Schottlaender, L., Lim, A., Palermo, C., Farrar, M., Janas, J., Laurent Servais, Butterfield, R., Vanlander, A., Sanabria, L, Wittevrongel, S., Kuntz, NL, Tahon, V., Jacqueline Montes, Jong, Y. J., Miller, N., Mizuochi, K., Palacios, N. J., Krosschell, K., Taniguchi, N., Kramer, S., Duong, T. T., Chau, V., Borell, S., Zilke, K., Már Tulinius, Ramamurthi, R. J., Ramamurthi, RJ, Mercuri, E., Pane, M., Montes, Jacqueline, Pasternak, A, Jachertz, P., Tizzano, E., Nakatsukasa, H., Nancy L. Kuntz, Koka, A., De Vivo, Darryl C, Stanford, D., Taytard, J., Servais, Laurent, Le Moing, A. G., Mercuri, Eugenio, Schroth, M. K., D'Argenzio, L., Haldenby, R., Gili, G., Cappell, J., Epinosa Garcia, S., Kaburagi, Y., Watson, K, Wittevrongel, Sylvia, Deconinck, Nicolas, Darras, B. T., Khandji, A., Glanzman, AM, Austin, E. D., Connolly, A. M., Sabapathy, C., Lee, J., Chadehumbe, M., Dinunzio, P., Finkel, Richard S, Watson, K., Koka, A, Gee, R., Zhong, Z. John, Pascual Pascual, S. I., Ross, A., Souris, M, Chiriboga, Claudia A, Bodensteriner, JB, Tizzano, Eduardo F., DeArmey, S, Lindstedt, A., Sponemann, N., Wu, Y. C., Davis, CS, Shichiji, M., Tizzano, E, Darras, BT, Darras, Basil T., Van der Looven, Ruth +564 morecore +2 more sourcesImplications of circulating neurofilamentsfor spinal muscular atrophytreatment early in life: A case series
Molecular Therapy: Methods & Clinical Development, 2021 This longitudinal cohort study aimed to determine whether circulating neurofilaments (NFs) can monitor response to molecular therapies in newborns with spinal muscular atrophy (SMA; NCT02831296).Christiano R.R. Alves, Marco Petrillo, Rebecca Spellman, Reid Garner, Ren Zhang, Michael Kiefer, Sarah Simeone, Jihee Sohn, Eric J. Eichelberger, Emma Rodrigues, Elizabeth A. Arruda, Elise L. Townsend, Wildon Farwell, Kathryn J. Swoboda +13 moredoaj +1 more sourceNusinersen in spinal muscular atrophy: Respiratory outcomes at tertiary care centers
Journal of Nature and Science of Medicine, 2020 Objectives: The objective of the study wasto describe the effect of nusinersen therapy and its 1st-year associated outcomes, weaning of ventilator support, and length of stay in pediatric intensive care among ventilator-dependent spinal muscular atrophy (Fahad Alsohime, Mohamad-Hani Temsah, Muneera Al-Jelaify, Hind Ali Bafaqih, Kamal El Masri, Ahmed Abusibah, Ayman Al-Eyadhy, Gamal Hasan, Ali Alhaboob, Majed Alabdulhafid, Mohammed Abdulrahman Almazyad, Abdulaziz Alghamdi, Fahad A Bashiri +12 moredoaj +1 more sourceNusinersen: A Review in 5q Spinal Muscular Atrophy
, 2021 DeclarationsFunding The preparation of this review was not supported by any external funding.Authorship and Conflict of interest Sheridan M. Hoy is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of ...Sheridan M. Hoy (4897468)core +1 more sourceThe Antisense Oligonucleotide Nusinersen for Treatment of Spinal Muscular Atrophy [PDF]
, 2021 Spinal muscular atrophy (SMA) is a rare, autosomal recessive neuromuscular degenerative disease characterized by loss of spinal cord motor neurons leading to progressive muscle wasting.John W. Pruitt, Adam M. Kaye, Girma, Brook, Pruitt, John W., Amber N. Edinoff, Cornett, Elyse M., Odisho, Amira S., Kaye, Alan David, Benjamin S. Maxey, Nguyen, Long H., Kaye, Adam M., Alan D. Kaye, Edinoff, Amber N, Amira S. Odisho, Elyse M. Cornett, Brook Girma, Maxey, Benjamin S., Long H. Nguyen +17 morecore +1 more sourceProxy-Reported Quality of Life and Access to Nusinersen Among Patients with Spinal Muscular Atrophy in Saudi Arabia
Patient Preference and Adherence, 2021 Yazed AlRuthia,1,2 Ghadah S Almuaythir,1 Hala H Alrasheed,1 Wejdan R Alsharif,1 Mohamad-Hani Temsah,3,4 Fahad Alsohime,3,4 Ibrahim Sales,1 Monira Alwhaibi,1 Fahad A Bashiri3,5 1Department of Clinical Pharmacy, College of Pharmacy, King Saud University ...AlRuthia Y, Almuaythir GS, H Alrasheed H, Alsharif WR, Temsah MH, Alsohime F, Sales I, Alwhaibi M, Bashiri FA +8 moredoaj Assessment of motor function and nutritional status in children with spinal muscular atrophy treated with nusinersen after loading period in Western China: a retrospective study
BMC Neurology, 2023 Background Spinal muscular atrophy (SMA) is a progressive degenerative neuromuscular disease. Nusinersen, with its quick onset of action, can benefit patients early in the treatment course.Hua Yang, Qiuji Tao, Dan Li, Jie Yang, Qianyun Cai, Jing Gan, Shaoping Huang, Rong Luo +7 moredoaj +1 more sourceCerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
Brain Sciences, 2021 Approval of nusinersen, an intrathecally administered antisense oligonucleotide, for the treatment of 5q-spinal muscular atrophy (SMA) marked the beginning of a new therapeutic era in neurological diseases.Lars Hendrik Müschen, Alma Osmanovic, Camilla Binz, Konstantin F. Jendretzky, Gresa Ranxha, Paul Bronzlik, Omar Abu-Fares, Flavia Wiehler, Nora Möhn, Martin W. Hümmert, Stefan Gingele, Friedrich Götz, Martin Stangel, Thomas Skripuletz, Olivia Schreiber-Katz, Susanne Petri +15 moredoaj +1 more source
