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P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study by Barbara Burton, Joseph Muenzer, Paul Harmatz, Deepa Rajan, Simon Jones, Johanna van den Hout, John Mitchell, Matthew Troyer, Natalie Engmann, Rupa Caprihan, Akhil Bhalla, Imanol Zubizarreta, Arlette Weichert, Peter Chin, Carole Ho
Published 2025-01-01Get full text
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Investigational eIF2B activator DNL343 modulates the integrated stress response in preclinical models of TDP-43 pathology and individuals with ALS in a randomized clinical trial by Brittany N. Flores, Seungyoon B. Yu, Isaac V. Cohen, Melania H. Fanok, Wei Luan, Romeo D. Maciuca, Linus D. Sun, Richard M. Tsai, Maurits Vissers, Lars Smits, Tommy M. Bunte, Anna Bakardjiev, Srijana Balasundar, Meredith E. K. Calvert, Marcus Y. Chin, Sarah K. Dobbins, William E. Dowdle, Meng Fang, Jules A. A. C. Heuberger, Connie L. Ha, Fen Huang, Takashi Miyamoto, Maksim Osipov, Lidia Madrid San Martin, Katie Saund, David Tatarakis, Anthony Q. Vu, Chenling Xiong, Gene W. Yeo, Geert Jan Groeneveld, Leonard H. van den Berg, Shyeilla Dhuria, Anthony A. Estrada, Danna Jennings, Thomas Sandmann, Carole Ho, Kimberly Scearce-Levie, Ernie Yulyaningsih, Adam K. Walker, Gilbert Di Paolo, Lesley A. Kane, Matthew D. Troyer, Joseph W. Lewcock
Published 2025-08-01Get full text
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