Stephanie Fradette

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

Darryl C. De Vivo, Enrico Bertini, Kathryn J. Swoboda et al. · Neuromuscular Disorders · 2019 · 626 citations · Full text

Medicine, Neurology, Pediatrics +10

Neurofilament as a potential biomarker for spinal muscular atrophy

Basil T. Darras, Thomas O. Crawford, Richard S. Finkel et al. · Annals of Clinical and Translational Neurology · 2019 · 207 citations · Full text

Medicine, Neurology, Neuroscience +17

Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study

Michael Benatar, Joanne Wuu, Peter M. Andersen et al. · Neurotherapeutics · 2022 · 166 citations · Full text

Life Sciences, Genetics, Neurology +17

Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases

Michael Benatar, Joanne Wuu, Caroline McHutchison et al. · Brain · 2021 · 116 citations · Full text

Life Sciences, Neurology, Neuroscience +15

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 et al. · The Lancet Neurology · 2024 · 70 citations · Full text

Life Sciences, Neurology, Pharmacokinetics +12