Concepedia

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

Richard S. Finkel, Claudia A. Chiriboga, Anne M. Connolly, Thomas O. Crawford, Basil T. Darras, Susan T. Iannaccone, Nancy L. Kuntz, Loren D.M. Peña, Perry B. Shieh, Edward C. Smith,

The Lancet Neurology · 2021 · 480 citations · 20 references

DOI

Concepts

References

20

WHO Child Growth Standards based on length/height, weight and age

Mercedes de Onís · Acta Paediatrica · 2006 · 4K citations

, , +17

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.3K citations · Full text

, , +16

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

Kevin D. Foust, Emily Nurre, Chrystal L. Montgomery et al. · Nature Biotechnology · 2008 · 1.4K citations · Full text

, , +8

Quantitative Analyses of SMN1 and SMN2 Based on Real-Time LightCycler PCR: Fast and Highly Reliable Carrier Testing and Prediction of Severity of Spinal Muscular Atrophy

Markus Feldkötter, Verena Schwarzer, Radu Wirth et al. · The American Journal of Human Genetics · 2002 · 1K citations · Full text

, , +8

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

DM McCarty, PE Monahan, RJ Samulski · Gene Therapy · 2001 · 725 citations

, , +12