Fellowship · La Jolla, CA
Sponsored by University of California (San Diego) Medical Center
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
The Lancet. Neurology · 2024 · 113 citations · Chamindra Laverty
Amyotrophic lateral sclerosis caused by hexanucleotide repeat expansions in C9orf72: from genetics to therapeutics.
The Lancet. Neurology · 2025 · 36 citations · John Ravits
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.
The Lancet. Neurology · 2024 · 71 citations · John Ravits
Heat-shock chaperone HSPB1 regulates cytoplasmic TDP-43 phase separation and liquid-to-gel transition.
Nature cell biology · 2022 · 137 citations · John Ravits
Neuronal aging causes mislocalization of splicing proteins and unchecked cellular stress.
Nature neuroscience · 2025 · 33 citations · John Ravits
Childhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis.
Nature medicine · 2021 · 135 citations · Chamindra Laverty
Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation.
Nature neuroscience · 2024 · 43 citations · John Ravits
Validity of the MoCA as a cognitive screening tool in epilepsy: Are there implications for global care and research?
Epilepsia open · 2024 · 16 citations · Vicente Iragui-Madoz