Fellowship · Richmond, VA
Sponsored by Virginia Commonwealth University Health System
Current clinical applications of AAV-mediated gene therapy.
Molecular therapy : the journal of the American Society of Gene Therapy · 2025 · 87 citations · Nicholas Johnson
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Neurology · 2024 · 70 citations · Amy Harper
Population-Based Prevalence of Myotonic Dystrophy Type 1 Using Genetic Analysis of Statewide Blood Screening Program.
Neurology · 2021 · 146 citations · Nicholas Johnson
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA neurology · 2022 · 114 citations · Amy Harper
The Splice Index as a prognostic biomarker of strength and function in myotonic dystrophy type 1.
The Journal of clinical investigation · 2025 · 24 citations · Nicholas Johnson
Tremor Syndromes: An Updated Review.
Frontiers in neurology · 2021 · 76 citations · Abhishek Lenka
An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1.
The New England journal of medicine · 2026 · 10 citations · Nicholas Johnson
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial.
The Lancet. Neurology · 2023 · 50 citations · Nicholas Johnson