Gene therapy - Wikipedia
How It Works | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors - ScienceDirect
Gene therapy successes point to better therapies | PNAS
Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy - Kichula - 2021 - Muscle & Nerve - Wiley Online Library
Spinal Muscular Atrophy | ASGCT - American Society of Gene & Cell Therapy
Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors - ScienceDirect
What is SMA? | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
IJMS | Free Full-Text | New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand? | HTML
Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients | Health And Medicine
Novartis' gene therapy Zolgensma gets Japan approval for neuromuscular disorder | S&P Global Market Intelligence
JCM | Free Full-Text | New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges | HTML
Oligonucleotides to the (Gene) Rescue: FDA Approvals 2017–2019: Trends in Pharmacological Sciences
Gene specific therapies – the next therapeutic milestone in neurology | Neurological Research and Practice | Full Text
SMA Gene Therapy is a Bargain - Even With a Price Tag of US $2.125 Million - BIO 501
How It Works | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Mechanism of action of nusinersen | Download Scientific Diagram
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text
Zolgensma for the Treatment of Spinal Muscular Atrophy, Novartis
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
Smart Nanocarriers for the Delivery of Nucleic Acid‐Based Therapeutics: A Comprehensive Review - Ramasamy - 2021 - Biotechnology Journal - Wiley Online Library
Advances in Treatments in Muscular Dystrophies and Motor Neuron Disorders - Neurologic Clinics
New Drug Product: Zolgensma - MPR
Zolgensma for the Treatment of Spinal Muscular Atrophy, Novartis
