Fellowship · San Diego, CA
Sponsored by University of California (San Diego) Medical Center
Skin Biopsy Detection of Phosphorylated α-Synuclein in Patients With Synucleinopathies.
JAMA · 2024 · 170 citations · Gregory Sahagian
Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study.
The Lancet. Neurology · 2023 · 236 citations · Gregory Sahagian
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 ·
Neuronal aging causes mislocalization of splicing proteins and unchecked cellular stress.
Nature neuroscience · 2025 · 33 citations · John Ravits
BCMA-directed mRNA CAR T cell therapy for myasthenia gravis: a randomized, double-blind, placebo-controlled phase 2b trial.
Nature medicine · 2026 · 14 citations · Gregory Sahagian