Results 151 to 160 of about 169,015 (342)
Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL
Muscle &Nerve, EarlyView.A total of 95 ERT‐experienced adults with LOPD were randomized to switch to cipaglucosidase alfa + miglustat or remain on alglucosidase alfa treatment. After 52 weeks, patients remaining on alglucosidase alfa showed worsening or stability for most outcomes, whereas patients who switched to cipaglucosidase alfa + miglustat generally showed stability or ...Hani Kushlaf, Jordi Díaz‐Manera, Drago Bratkovic, Barry J. Byrne, Kristl G. Claeys, Paula R. Clemens, Mazen M. Dimachkie, Priya S. Kishnani, Pascal Laforêt, Mark Roberts, Benedikt Schoser, Antonio Toscano, Jeff Castelli, Fred Holdbrook, Sheela Sitaraman Das, Mitchell Goldman, Tahseen Mozaffar, on behalf of the PROPEL Study Group +17 morewiley +1 more sourceOperational Development and Launch of an Adaptive Platform Trial in Amyotrophic Lateral Sclerosis: Processes and Learnings From the First Four Regimens of the HEALEY ALS Platform Trial
Muscle &Nerve, EarlyView.ABSTRACT Introduction/Aims
Platform trials present several advantages over traditional interventional clinical trials. Here, we provide a detailed description of the operational framework of the HEALEY ALS Platform Trial. Methods
Platform‐level procedures for regulatory oversight, safety, and site management were developed prior to trial launch ...Brittney A. Harkey, Sofia Distefano, Jaclyn A. Pagliaro, Lindsay Heyd, Marianne Chase, Courtney Igne, Hong Yu, Alexander V. Sherman, Derek Dagostino, Eric Tustison, Genevive Changkuon, Meghan Hall, Gale Kittle, Mariah R. Connolly, Elisa Giacomelli, Erica Scirocco, James D. Berry, Suma Babu, Jeremy Shefner, Eric A. Macklin, Lori B. Chibnik, Annette De Mattos, Kristin Drake, Cornelia Kamp, Andrew McGarry, Margherita Torti, Catherine Small, Allison Bulat, Merit E. Cudkowicz, Sabrina Paganoni, for the HEALEY ALS Platform Trial Study Group, Eric A. Macklin, Lori B. Chibnik, Douglas Hayden, Po‐Ying Lai, Rachel A. Donahue, Hao‐Wun Chen, Jianing Wang, Melanie Quintana, Benjamin R. Saville, Michelle A. Detry, Joe Marion, Matteo Vestrucci, Anna McGlothlin, Gustavo Alameda, Nithya Mathai, Eduardo Locatelli, Gabriela Lopes, Sabrina Paganoni, Merit Cudkowicz, Doreen Ho, Alexandra McCaffrey, James D. Berry, Suma Babu, Jennifer Scalia, Sarah Luppino, Clotilde Lagier‐Tourenne, Ghazaleh Sadri‐Vakili, Elisa Giacomelli, Erica Scirocco, Raina Chrobak, Adam Quick, Stephen Kolb, Sarah Heintzman, Senda Ajroud‐Driss, Robert Sufit, April Szymanski, Jonathan Katz, Liberty Jenkins, Daragh Heitzman, Alan Martin, Stanley H. Appel, (Simpson) Greene Ericka, Jason R. Thonhoff, Sheetal Shroff, Bing Liao, Kevin Felice, Charles Whitaker, Nicholas J. Maragakis, Lora L. Clawson, Alpa Uchil, Kristen M. Riley, JinAe Arneklev, Zachary Simmons, James Grogan, Xiaowei Su, Mansoureh Mamarabadi, Timothy M. Miller, Amber Malcolm, Nicholas Olney, Tracy Bazan, Michael D Weiss, Nassim Rad, Leo H. Wang, Stephen A. Goutman, Eva L. Feldman, Joseph Americo Fernandes, Ezequiel Piccione, Pariwat Thaisetthawatkul, Omar Jawdat, Constantine Farmakidis, Duaa Jabari, Jeffrey Statland, Mamatha Pasnoor, Mazen Dimachkie, Margaret Ayo Owegi, Robert H. Brown, Mehdi Ghasemi, Hajar Houmani, Catherine Douthwright, Kate Daniello, Laura A. Foster, Tuan Vu, Niraja Suresh, Jerrica Farias, I‐Hweii A. Chen, Hristelina Ilieva, Piera Pasinelli, Daniel S. Newman, Ximena Arcila‐Londono, Kara Steijlen, Carlayne E. Jackson MD, Ratna Bhavaraju‐Sanka, Shafeeq Ladha, Bill Jacobsen, Jourdan Milliard, Robert Bowser, Jeremy M. Shefner, Terry Heiman‐Patterson, Anahita Deboo, James B. Caress, Michael S. Cartwright, Andrea Swenson, Christopher Nance, Ludwig Gutmann, Jinsy Andrews, Julia Yasek, Matthew Harms, Amanda Peltier, Richard Lewis, Matthew Burford, Frank Diaz, Dominic Fee, David Shrilla, Matthew Elliott, Goran Rakocevic, Sarah Jones, Guillermo Solorzano, Richard Bedlack, Xiaoyan Li, Edward J. Kasarskis, Zabeen Mahuwala, (Vish) Mathur Kumaraswamy Vishakhadatta, MD Lauren Elman, Colin Quinn, Michael Baer, Jeffrey Rosenfeld, David Borg, Karthikeyan Bhuvaneswaran, Jasdeep Kaur, David Walk, Sam Maiser, Seward B. Rutkove, Courtney E. McIlduff, Paul Twydell, Andrew Mundwiler, Eufrosina Young, Jenny A. Meyer, Kristin Johnson, Pooja Rao, Kourosh Rezania, Betty Soliven, Raymond Roos, Namita A. Goyal, Ali A. Habib, Tahseen Mozaffar, Manisha Kak Korb, Jeffrey Mullen, Jeffrey A. Cohen, Elijah Stommel, Nathaniel M Robbins, Michael Benatar, Nathan Carberry, Volkan Granit, Raghav Govindarajan, Vovanti Jones, Christina N. Fournier, Jaimin S. Shah, Bjorn Oskarsson, Said R. Beydoun, Leila Darki, Rodrigo Rodriguez, James P. Wymer, Miguel Chuquilin, Whitney McNeely, Lindsay Zilliox, Montserrat Diaz‐Abad, Peter H. Jin, Chandana Chauhan, Shakti Nayar, Gary L. Pattee, James Bobenhouse, Jennifer Martinez‐Thompson, P. Staff Nathan, Ghazala Hayat, Luisa Arroyave, Abbey Bailey, Jesse Bailey, Victoria Barlow, Allison Bulat, Genevive Changkuon, Marianne Chase, Melissa Cirino, Derek Dagostino, Cristina Deignan, Emma Deirmendjian, Annette De Mattos, Sofia DiStefano, Kristin Drake, Michaela Estes, Kenneth Faulconer, Precious Figueroa‐Szostek, Tessa Garozzo, Brittney A. Harkey, Meredith Gibbons Hasenoehrl, Jennifer Henrique, Natalie Henrique, Samuel Hurwitz, Courtney Igne, Liam Irwin, Katie Jentoft, Boglarka Jordan, Igor Katsovskiy, Olga Kharakozova, Taylor Kolvek, Alexander Korin, Thuong La, Haining Li, Joey Nguyen, Ilya Novak, Ricardo Ortiz, Joe Ostrow, Jaclyn Pagliaro, Jack Palillo, Payal Patel, Janae Patterson, Minh Phan, Najla Popel, Lindsay Pothier, Serena Proueng, Jesse Rosenthal, Alexander V. Sherman, Catherine Small, Natalia Tarasenko, Mirna Thomas, Eric Tustison, Prasha Vigneswaran, Yusra Wahab, Isaac Whitworth, Spencer Wright, Hong Yu, Mariah Connolly, Diana De Santiago, Adrian Felix, Karly Garrett, Meghan Hall, Jenny Hamilton, Kamran Khan, Gale Kittle, Marlee Lovett, Linda Nelson, Marissa Pabon, Diana Rede, Patrick Bolger, Ahmed Fetouh, Joan Woodcook, Cornelia Kamp, Julie Kennedy, Andrew McGarry, Margherita Torti +279 morewiley +1 more sourceA Phase 1, Double‐Blind, Placebo‐Controlled Trial of Sevasemten (EDG‐5506), a Selective Modulator of Fast Skeletal Muscle Contraction, in Healthy Volunteers and Adults With Becker Muscular Dystrophy
Muscle &Nerve, EarlyView.ABSTRACT Introduction/Aims
Sevasemten (EDG‐5506) is an orally administered, investigational small molecule that selectively modulates fast muscle fiber contraction by inhibiting fast myosin ATPase. This study assessed the safety, tolerability, and pharmacokinetics (PK)/pharmacodynamics (PD) of sevasemten in healthy adult volunteers (HVs) and adults ...Joanne Donovan, Jeffrey A. Silverman, Ben Barthel, Michael DuVall, Molly Madden, James MacDougall, Nicole Rempel Kilburn, Abby Bronson, Marc Evanchik, Gilad Gordon, Kevin Koch, Alan J Russell +11 morewiley +1 more sourcePlacebo‐Controlled, Randomized Double‐Blind N‐Of‐1 Trial to Study Safety and Potential Efficacy of TJ‐68 for Improving Muscle Cramps in Patients With Amyotrophic Lateral Sclerosis: A Pilot Study
Muscle &Nerve, EarlyView.ABSTRACT Introduction/Aims
Muscle cramps are a common symptom in amyotrophic lateral sclerosis (ALS). Ameliorating muscle cramps may improve quality of life in devastating diseases like ALS. A traditional Japanese medicine (Kampo, TJ‐68) is widely prescribed in Japan for muscle cramps. However, it is not available in the USA.Hiroshi Mitsumoto, Ken Cheung, Björn Oskarsson, Grace E. Jang, Howard F. Andrews, Stephen Johnson, Jaimin S. Shah, Joseph Americo Fernandes Jr, Jinsy A. Andrews, Maya Rao, Martin McElhiney +10 morewiley +1 more source
