Nusinersen Treatment of Children with Later-Onset Spinal Muscular Atrophy and Scoliosis Is Associated with Improvements or Stabilization of Motor Function
Dunaway Young, Sally, Montes, Jacqueline, Glanzman, Allan M, Gee, Richard, Day, John W, Finkel, Richard S, Darras, Basil T, De Vivo, Darryl C, Gambino, Giulia, Foster, Richard, Wong, Janice, Garafalo, Steve, Berger, Zdenek
Published in Journal of clinical medicine (26.07.2023)
Published in Journal of clinical medicine (26.07.2023)
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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
van den Berg, Leonard H, Rothstein, Jeffrey D, Shaw, Pamela J, Babu, Suma, Benatar, Michael, Bucelli, Robert C, Genge, Angela, Glass, Jonathan D, Hardiman, Orla, Libri, Vincenzo, Mobach, Theodore, Oskarsson, Björn, Pattee, Gary L, Ravits, John, Shaw, Christopher E, Weber, Markus, Zinman, Lorne, Jafar-nejad, Paymaan, Rigo, Frank, Lin, Luan, Ferguson, Toby A, Gotter, Anthony L, Graham, Danielle, Monine, Michael, Inra, Jennifer, Sinks, Susie, Eraly, Satish, Garafalo, Steve, Fradette, Stephanie
Published in Lancet neurology (01.09.2024)
Published in Lancet neurology (01.09.2024)
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Personalized medicines in late-stage development
Milne, Christopher-Paul, Garafalo, Steve, Bryan, Crystal, McKiernan, Marie
Published in Nature reviews. Drug discovery (01.05.2014)
Published in Nature reviews. Drug discovery (01.05.2014)
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Trial watch: personalized medicines in late-stage development
Milne, Christopher-Paul, Garafalo, Steve, Bryan, Crystal, McKiernan, Marie
Published in Nature reviews. Drug discovery (01.05.2014)
Published in Nature reviews. Drug discovery (01.05.2014)
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