Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study

著者: Leonard H. van den Berg, Jeffrey D. Rothstein, Pamela J. Shaw, Suma Babu, Michael Benatar, Robert C. Bucelli, Angela Genge, Jonathan D. Glass, Orla Hardiman, Vincenzo Libri, Theodore Mobach, Björn Oskarsson, Gary L. Pattee, John Ravits, Christopher E. Shaw, Markus Weber, Lorne Zinman, Paymaan Jafar‐Nejad, Frank Rigo, Luan Lin, Toby A. Ferguson, Anthony L. Gotter, Danielle Graham, Michael Monine, Jennifer Inra, Susie Sinks, Satish A. Eraly, Steve Garafalo, Stephanie Fradette - The Lancet 2024 被引用: 70