Safety and tolerability of BG-12 (dimethyl fumarate) in relapsing–/INS;remitting multiple sclerosis: Interim results from the endorse extension study von Selmaj, K, Phillips, J.T, Fox, R.J, Raghupathi, K, Yuan, H, Novas, M, Sweetser, M.T, Kurukulasuriya, N.C, Viglietta, V, Dawson, K.T, Gold, R

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BG-12 (dimethyl fumarate) and pregnancy: Preclinical studies and pregnancy outcomes reported during the clinical development program von Gold, R, Phillips, J.T, Havrdova, E, Bar-Or, A, Kappos, L, Clarke, J, Yuan, H, Novas, M, Sweetser, M.T, Kurukulasuriya, N.C, Viglietta, V, Fox, R.J

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Long-term integrated safety of patisiran in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy von Gillmore, J, Berk, J, Dispenzieri, A, Polydefkis, M, Gonzalez-Duarte, A, Sekijima, Y, Sweetser, M.T, Arum, S, Wang, J.J, White, M.T, Maurer, M

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Patisiran treatment for ATTR cardiac amyloidosis: 18-month results of the phase 3 APOLLO-B study von Fontana, M., L Berk, J., Hanna, M., D Gillmore, J., Pedro-Schwartzmann, V., Lairez, O., Hyun-Jai, C., Van Der Meer, P., White, M.T., Elena, Y., Sweetser, M.T., Jay, P.Y., Vest, J., S Maurer, M.

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Risk Factors for Mortality in Patients with Hereditary Transthyretin-Mediated Amyloidosis: An Analysis of APOLLO and Global Open Label Extension Studies von Polydefkis, M., Gillmore, J., Dispenzieri, A., Chen, J., Sweetser, M.T., Vest, J., Melanson, M., Conceicao, I., Kristen, A.

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Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis von Maurer, Mathew S., Kale, Parag, Fontana, Marianna, Berk, John L., Grogan, Martha, Gustafsson, Finn, Hung, Rebecca R., Gottlieb, Robert L., Damy, Thibaud, González-Duarte, Alejandra, Sarswat, Nitasha, Sekijima, Yoshiki, Tahara, Nobuhiro, Taylor, Mark S., Kubanek, Milos, Donal, Erwan, Palecek, Tomas, Tsujita, Kenichi, Tang, W.H. Wilson, Yu, Wen-Chung, Obici, Laura, Simões, Marcus, Fernandes, Fábio, Poulsen, Steen Hvitfeldt, Diemberger, Igor, Perfetto, Federico, Solomon, Scott D., Di Carli, Marcelo, Badri, Prajakta, White, Matthew T., Chen, Jihong, Yureneva, Elena, Sweetser, Marianne T., Jay, Patrick Y., Garg, Pushkal P., Vest, John, Gillmore, Julian D.

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Résultats primaires de l’étude APOLLO-B d’extension en ouvert du patisiran chez les patients atteints d’amylose cardiaque à transthyrétine von Maurer, M.S., Berk, J.L., Gillmore, J.D., Lairez, O., White, M.T., Sweetser, M., Lainé, A.F., Fontana, M.

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A Recurrent De Novo PACS2 Heterozygous Missense Variant Causes Neonatal-Onset Developmental Epileptic Encephalopathy, Facial Dysmorphism, and Cerebellar Dysgenesis von Olson, H.E., Jean-Marcais, N., Yang, E., Heron, D., Tatton-Brown, K., Zwaag, P.A. van der, Bijlsma, E.K., Krock, B.L., Backer, E., Kamsteeg, E.J., Sinnema, M., Reijnders, M.R.F., Bearden, D., Begtrup, A., Telegrafi, A., Lunsing, R.J., Burglen, L., Lesca, G., Cho, M.T., Smith, L.A., Sheidley, B.R., Achkar, C.M. el, Pearl, P.L., Poduri, A., Skraban, C.M., Tarpinian, J., Nesbitt, A.I., Putte, D.E.F. van de, Ruivenkamp, C.A.L., Rump, P., Chatron, N., Sabatier, I., Bellescize, J. de, Guibaud, L., Sweetser, D.A., Waxler, J.L., Wierenga, K.J., Donadieu, J., Narayanan, V., Ramsey, K.M., Nava, C., Riviere, J.B., Vitobello, A., Mau-Them, F.T., Philippe, C., Bruel, A.L., Duffourd, Y., Thomas, L., Lelieveld, S.H., Schuurs-Hoeijmakers, J., Brunner, H.G., Keren, B., Thevenon, J., Faivre, L., Thomas, G., Thauvin-Robinet, C.

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