Fellowship · St Louis, MO
Sponsored by Washington University/B-JH/SLCH Consortium
Guidelines for the use and interpretation of assays for monitoring autophagy (4th edition)1.
Autophagy · 2021 · 2,291 citations · Conrad Weihl
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
The New England journal of medicine · 2022 · 755 citations · Timothy Miller, Robert Bucelli
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Nature medicine · 2025 · 143 citations · Craig Zaidman
Skin Biopsy Detection of Phosphorylated α-Synuclein in Patients With Synucleinopathies.
JAMA · 2024 · 170 citations · Todd Levine
Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.
The Lancet. Neurology · 2023 · 218 citations · Yessar Hussain
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
The Lancet. Neurology · 2021 · 399 citations ·
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Nature medicine · 2022 · 263 citations · Jennifer Kwon, Craig Zaidman
A fluid biomarker reveals loss of TDP-43 splicing repression in presymptomatic ALS-FTD.
Nature medicine · 2024 · 142 citations · Mark Garret, Timothy Miller