The once and future gene therapy | Nature Communications
How It Works | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Advances in Treatment of Spinal Muscular Atrophy – New Phenotypes, New Challenges, New Implications for Care - IOS Press
Spinal muscular atrophy — insights and challenges in the treatment era | Nature Reviews Neurology
How It Works | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Zolgensma for the Treatment of Spinal Muscular Atrophy, Novartis
Viral vector platforms within the gene therapy landscape | Signal Transduction and Targeted Therapy
Maybe too much of a good thing in gene therapy | Nature Neuroscience
Frontiers | Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment | Pharmacology
Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors - ScienceDirect
What is SMA? | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
MOA | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Spinraza switches splicing to save patients with Spinal Muscular Atrophy (SMA)… some cool science to know about this ASO! (and other SMA treatments) – The Bumbling Biochemist
Zolgensma for the Treatment of Spinal Muscular Atrophy, Novartis
Smart Nanocarriers for the Delivery of Nucleic Acid‐Based Therapeutics: A Comprehensive Review - Ramasamy - 2021 - Biotechnology Journal - Wiley Online Library
Spinal Muscular Atrophy | ASGCT - American Society of Gene & Cell Therapy
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text
Gene therapy successes point to better therapies | PNAS
Onasemnogene Abeparvovec - an overview | ScienceDirect Topics
Zolgensma for the Treatment of Spinal Muscular Atrophy, Novartis
