Immune Modulation in the Treatment of Amyotrophic Lateral Sclerosis: A Review of Clinical Trials

por Khalid, Syed I., Ampie, Leonel, Kelly, Ryan, Ladha, Shafeeq S., Dardis, Christopher
Publicado 2017

Evaluation of Amyotrophic Lateral Sclerosis-Induced Muscle Degeneration Using Magnetic Resonance-Based Relaxivity Contrast Imaging (RCI)

por Ragunathan, Sudarshan, Bell, Laura C., Semmineh, Natenael, Stokes, Ashley M., Shefner, Jeremy M., Bowser, Robert, Ladha, Shafeeq, Quarles, C. Chad
Publicado 2021

Longitudinal biomarkers in amyotrophic lateral sclerosis

por Huang, Fen, Zhu, Yuda, Hsiao‐Nakamoto, Jennifer, Tang, Xinyan, Dugas, Jason C., Moscovitch‐Lopatin, Miriam, Glass, Jonathan D., Brown, Robert H., Ladha, Shafeeq S., Lacomis, David, Harris, Jeffrey M., Scearce‐Levie, Kimberly, Ho, Carole, Bowser, Robert, Berry, James D.
Publicado 2020

Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD

por Krishnan, Gopinath, Raitcheva, Denitza, Bartlett, Daniel, Prudencio, Mercedes, McKenna-Yasek, Diane M., Douthwright, Catherine, Oskarsson, Björn E., Ladha, Shafeeq, King, Oliver D., Barmada, Sami J., Miller, Timothy M., Bowser, Robert, Watts, Jonathan K., Petrucelli, Leonard, Brown, Robert H., Kankel, Mark W., Gao, Fen-Biao
Publicado 2022

Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease

por Dimachkie, Mazen M., Barohn, Richard J., Byrne, Barry, Goker-Alpan, Ozlem, Kishnani, Priya S., Ladha, Shafeeq, Laforêt, Pascal, Mengel, Karl Eugen, Peña, Loren D.M., Sacconi, Sabrina, Straub, Volker, Trivedi, Jaya, Van Damme, Philip, van der Ploeg, Ans T., Vissing, John, Young, Peter, Haack, Kristina An, Foster, Meredith, Gilbert, Jane M., Miossec, Patrick, Vitse, Olivier, Zhou, Tianyue, Schoser, Benedikt
Publicado 2022

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial

por Kishnani, Priya S., Diaz-Manera, Jordi, Toscano, Antonio, Clemens, Paula R., Ladha, Shafeeq, Berger, Kenneth I., Kushlaf, Hani, Straub, Volker, Carvalho, Gerson, Mozaffar, Tahseen, Roberts, Mark, Attarian, Shahram, Chien, Yin-Hsiu, Choi, Young-Chul, Day, John W., Erdem-Ozdamar, Sevim, Illarioshkin, Sergey, Goker-Alpan, Ozlem, Kostera-Pruszczyk, Anna, van der Ploeg, Ans T., An Haack, Kristina, Huynh-Ba, Olivier, Tammireddy, Swathi, Thibault, Nathan, Zhou, Tianyue, Dimachkie, Mazen M., Schoser, Benedikt
Publicado 2023

Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR tria...

por Paganoni, Sabrina, Hendrix, Suzanne, Dickson, Samuel P, Knowlton, Newman, Berry, James D, Elliott, Michael A, Maiser, Samuel, Karam, Chafic, Caress, James B, Owegi, Margaret Ayo, Quick, Adam, Wymer, James, Goutman, Stephen A, Heitzman, Daragh, Heiman-Patterson, Terry D, Jackson, Carlayne, Quinn, Colin, Rothstein, Jeffrey D, Kasarskis, Edward J, Katz, Jonathan, Jenkins, Liberty, Ladha, Shafeeq S, Miller, Timothy M, Scelsa, Stephen N, Vu, Tuan H, Fournier, Christina, Johnson, Kristin M, Swenson, Andrea, Goyal, Namita, Pattee, Gary L, Babu, Suma, Chase, Marianne, Dagostino, Derek, Hall, Meghan, Kittle, Gale, Eydinov, Mathew, Ostrow, Joseph, Pothier, Lindsay, Randall, Rebecca, Shefner, Jeremy M, Sherman, Alexander V, Tustison, Eric, Vigneswaran, Prasha, Yu, Hong, Cohen, Joshua, Klee, Justin, Tanzi, Rudolph, Gilbert, Walter, Yeramian, Patrick, Cudkowicz, Merit
Publicado 2022

A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS

por SHEFNER, JEREMY M., ANDREWS, JINSY A., GENGE, ANGELA, JACKSON, CARLAYNE, LECHTZIN, NOAH, MILLER, TIMOTHY M., COCKROFT, BETTINA M., MENG, LISA, WEI, JENNY, WOLFF, ANDREW A., MALIK, FADY I., BODKIN, CYNTHIA, BROOKS, BENJAMIN R., CARESS, JAMES, DIONNE, ANNIE, FEE, DOMINIC, GOUTMAN, STEPHEN A., GOYAL, NAMITA A., HARDIMAN, ORLA, HAYAT, GHAZALA, HEIMAN-PATTERSON, TERRY, HEITZMAN, DARAGH, HENDERSON, ROBERT D., JOHNSTON, WENDY, KARAM, CHAFIC, KIERNAN, MATTHEW C., KOLB, STEPHEN J., KORNGUT, LAWRENCE, LADHA, SHAFEEQ, MATTE, GENEVIEVE, MORA, JESUS S., NEEDHAM, MERRILEE, OSKARSSON, BJORN, PATTEE, GARY L., PIORO, ERIK P., PULLEY, MICHAEL, QUAN, DIANNA, REZANIA, KOUROSH, SCHELLENBERG, KERRI L., SCHULTZ, DAVID, SHOESMITH, CHRISTEN, SIMMONS, ZACHARY, STATLAND, JEFFREY, SULTAN, SHUMAILA, SWENSON, ANDREA, VAN DEN BERG, LEONARD H., VU, TUAN, VUCIC, STEVE, WEISS, MICHAEL, WHYTE-RAYSON, ASHLEY, WYMER, JAMES, ZINMAN, LORNE, RUDNICKI, STACY A.
Publicado 2020

Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosis

por Paganoni, Sabrina, Hendrix, Suzanne, Dickson, Samuel P., Knowlton, Newman, Macklin, Eric A., Berry, James D., Elliott, Michael A., Maiser, Samuel, Karam, Chafic, Caress, James B., Owegi, Margaret Ayo, Quick, Adam, Wymer, James, Goutman, Stephen A., Heitzman, Daragh, Heiman‐Patterson, Terry D., Jackson, Carlayne E., Quinn, Colin, Rothstein, Jeffrey D., Kasarskis, Edward J., Katz, Jonathan, Jenkins, Liberty, Ladha, Shafeeq, Miller, Timothy M., Scelsa, Stephen N., Vu, Tuan H., Fournier, Christina N., Glass, Jonathan D., Johnson, Kristin M., Swenson, Andrea, Goyal, Namita A., Pattee, Gary L., Andres, Patricia L., Babu, Suma, Chase, Marianne, Dagostino, Derek, Hall, Meghan, Kittle, Gale, Eydinov, Matthew, McGovern, Michelle, Ostrow, Joseph, Pothier, Lindsay, Randall, Rebecca, Shefner, Jeremy M., Sherman, Alexander V., St Pierre, Maria E., Tustison, Eric, Vigneswaran, Prasha, Walker, Jason, Yu, Hong, Chan, James, Wittes, Janet, Yu, Zi‐Fan, Cohen, Joshua, Klee, Justin, Leslie, Kent, Tanzi, Rudolph E., Gilbert, Walter, Yeramian, Patrick D., Schoenfeld, David, Cudkowicz, Merit E.
Publicado 2020