Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure‐Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin‐Mediated (hATTR) Amyloidosis

por Zhang, Xiaoping, Goel, Varun, Attarwala, Husain, Sweetser, Marianne T., Clausen, Valerie A., Robbie, Gabriel J.
Publicado 2019

Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study

por Wang, Bruce, Ventura, Paolo, Takase, Kei-ichiro, Thapar, Manish, Cassiman, David, Kubisch, Ilja, Liu, Shangbin, Sweetser, Marianne T., Balwani, Manisha
Publicado 2022

Cutaneous Adverse Events in the Randomized, Double-Blind, Active-Comparator DECIDE Study of Daclizumab High-Yield Process Versus Intramuscular Interferon Beta-1a in Relapsing-Remit...

por Krueger, James G., Kircik, Leon, Hougeir, Firas, Friedman, Adam, You, Xiaojun, Lucas, Nisha, Greenberg, Steven J., Sweetser, Marianne, Castro-Borrero, Wanda, McCroskery, Peter, Elkins, Jacob
Publicado 2016

EXPLORE B: A prospective, long‐term natural history study of patients with acute hepatic porphyria with chronic symptoms

por Cassiman, David, Kauppinen, Raili, Monroy, Susana, Lee, Ming‐Jen, Bonkovsky, Herbert L., Thapar, Manish, Guillén‐Navarro, Encarna, Minder, Anna‐Elisabeth, Hale, Cecilia, Sweetser, Marianne T., Ivanova, Aneta
Publicado 2022

Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial

por Hayes, Wesley, Sas, David J., Magen, Daniella, Shasha-Lavsky, Hadas, Michael, Mini, Sellier-Leclerc, Anne-Laure, Hogan, Julien, Ngo, Taylor, Sweetser, Marianne T., Gansner, John M., McGregor, Tracy L., Frishberg, Yaacov
Publicado 2022

Delayed-Release Dimethyl Fumarate and Pregnancy: Preclinical Studies and Pregnancy Outcomes from Clinical Trials and Postmarketing Experience

por Gold, Ralf, Phillips, J. Theodore, Havrdova, Eva, Bar-Or, Amit, Kappos, Ludwig, Kim, Norman, Thullen, Tim, Valencia, Patricia, Oliva, Lauren, Novas, Mark, Li, Jie, Sweetser, Marianne T., Kurukulasuriya, Nuwan, Viglietta, Vissia, Fox, Robert J.
Publicado 2015

A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

por Coelho, Teresa, Adams, David, Conceição, Isabel, Waddington-Cruz, Márcia, Schmidt, Hartmut H., Buades, Juan, Campistol, Josep, Berk, John L., Polydefkis, Michael, Wang, Jing Jing, Chen, Jihong, Sweetser, Marianne T., Gollob, Jared, Suhr, Ole B.
Publicado 2020

Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR)

por Judge, Daniel P., Kristen, Arnt V., Grogan, Martha, Maurer, Mathew S., Falk, Rodney H., Hanna, Mazen, Gillmore, Julian, Garg, Pushkal, Vaishnaw, Akshay K., Harrop, Jamie, Powell, Christine, Karsten, Verena, Zhang, Xiaoping, Sweetser, Marianne T., Vest, John, Hawkins, Philip N.
Publicado 2020

Efficacy and safety of givosiran for acute hepatic porphyria: 24‐month interim analysis of the randomized phase 3 ENVISION study

por Ventura, Paolo, Bonkovsky, Herbert L., Gouya, Laurent, Aguilera‐Peiró, Paula, Montgomery Bissell, D., Stein, Penelope E., Balwani, Manisha, Anderson, D. Karl E., Parker, Charles, Kuter, David J., Monroy, Susana, Oh, Jeeyoung, Ritchie, Bruce, Ko, John J., Hua, Zhaowei, Sweetser, Marianne T., Sardh, Eliane
Publicado 2021

Long-term effects of delayed-release dimethyl fumarate in multiple sclerosis: Interim analysis of ENDORSE, a randomized extension study

por Gold, Ralf, Arnold, Douglas L, Bar-Or, Amit, Hutchinson, Michael, Kappos, Ludwig, Havrdova, Eva, MacManus, David G, Yousry, Tarek A, Pozzilli, Carlo, Selmaj, Krysztof, Sweetser, Marianne T, Zhang, Ray, Yang, Minhua, Potts, James, Novas, Mark, Miller, David H, Kurukulasuriya, Nuwan C, Fox, Robert J, Phillips, Theodore J
Publicado 2016

Correction to: Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

por González-Duarte, Alejandra, Berk, John L., Quan, Dianna, Mauermann, Michelle L., Schmidt, Hartmut H., Polydefkis, Michael, Waddington-Cruz, Márcia, Ueda, Mitsuharu, Conceição, Isabel M., Kristen, Arnt V., Coelho, Teresa, Cauquil, Cécile A., Tard, Céline, Merkel, Madeline, Aldinc, Emre, Chen, Jihong, Sweetser, Marianne T., Wang, Jing Jing, Adams, David
Publicado 2020

Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

por González-Duarte, Alejandra, Berk, John L., Quan, Dianna, Mauermann, Michelle L., Schmidt, Hartmut H., Polydefkis, Michael, Waddington-Cruz, Márcia, Ueda, Mitsuharu, Conceição, Isabel M., Kristen, Arnt V., Coelho, Teresa, Cauquil, Cécile A., Tard, Céline, Merkel, Madeline, Aldinc, Emre, Chen, Jihong, Sweetser, Marianne T., Wang, Jing Jing, Adams, David
Publicado 2019

Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1

por Hulton, Sally A., Groothoff, Jaap W., Frishberg, Yaacov, Koren, Michael J., Overcash, J. Scott, Sellier-Leclerc, Anne-Laure, Shasha-Lavsky, Hadas, Saland, Jeffrey M., Hayes, Wesley, Magen, Daniella, Moochhala, Shabbir H., Coenen, Martin, Simkova, Eva, Garrelfs, Sander F., Sas, David J., Meliambro, Kristin A., Ngo, Taylor, Sweetser, Marianne T., Habtemariam, Bahru A., Gansner, John M., McGregor, Tracy L., Lieske, John C.
Publicado 2021