Fellowship · Boston, MA
Sponsored by Mass General Brigham
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
The New England journal of medicine · 2022 · 755 citations · Suma Babu
Spinal muscular atrophy.
Nature reviews. Disease primers · 2022 · 355 citations · Basil Darras
A Clinical Practice Guideline for the Management of Patients With Acute Spinal Cord Injury: Recommendations on Hemodynamic Management.
Global spine journal · 2024 · 85 citations · Lindsay Tetreault
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 · Basil Darras, Perry Shieh
Risdiplam in Type 1 Spinal Muscular Atrophy.
The New England journal of medicine · 2021 · 347 citations · Basil Darras
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 ·
A fluid biomarker reveals loss of TDP-43 splicing repression in presymptomatic ALS-FTD.
Nature medicine · 2024 · 142 citations · James Berry
An Update of a Clinical Practice Guideline for the Management of Patients With Acute Spinal Cord Injury: Recommendations on the Role and Timing of Decompressive Surgery.
Global spine journal · 2024 · 69 citations · Lindsay Tetreault