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
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
Topics & Concepts
Amyotrophic lateral sclerosisTolerabilityPharmacokineticsPlaceboMedicineC9orf72Internal medicinePharmacologyAdverse effectPathologyDiseaseDementiaAlternative medicineFrontotemporal dementiaAmyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders ResearchGenetic Neurodegenerative Diseases