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

Hexanucleotide repeat expansion of C9orf72 is a common genetic cause of amyotrophic lateral sclerosis (ALS). No C9orf72-targeted treatments are available. BIIB078 is an investigational antisense oligonucleotide targeting C9orf72 sense RNA. We aimed to assess the safety, tolerability, and pharmacokin...

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Veröffentlicht in:	Lancet neurology 2024-09, Vol.23 (9), p.901-912
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Adverse events Aged Amyotrophic lateral sclerosis Amyotrophic Lateral Sclerosis - drug therapy Amyotrophic Lateral Sclerosis - genetics Antisense oligonucleotides Antisense RNA Antisense therapy C9orf72 Protein - genetics Dose-Response Relationship, Drug Double-Blind Method Embolism Female Hemorrhage Humans Male Middle Aged Oligonucleotides, Antisense - administration & dosage Oligonucleotides, Antisense - adverse effects Oligonucleotides, Antisense - pharmacokinetics Oligonucleotides, Antisense - pharmacology Ostomy Patients Pharmacokinetics Placebos Proteins
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