SMA Gene Therapy is a Bargain - Even With a Price Tag of US $2.125 Million - BIO 501
Gene specific therapies – the next therapeutic milestone in neurology | Neurological Research and Practice | Full Text
Frontiers | Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment | Pharmacology
Potential mechanism of action of risdiplam. Risdiplam analogs SMN-C3... | Download Scientific Diagram
IJMS | Free Full-Text | New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand? | HTML
Mechanisms of action of main molecular therapeutic strategies that are... | Download Scientific Diagram
Zolgensma: World's Most Expensive Drug for SMA - All you need to know - Medinaz Blog
JCM | Free Full-Text | New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges | HTML
Spinal muscular atrophy — insights and challenges in the treatment era | Nature Reviews Neurology
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
Zolgensma for the Treatment of Spinal Muscular Atrophy, Novartis
The approved gene therapy drugs worldwide: from 1998 to 2019 - ScienceDirect
Zolgensma for the Treatment of Spinal Muscular Atrophy, Novartis
Novartis' gene therapy Zolgensma gets Japan approval for neuromuscular disorder | S&P Global Market Intelligence
How It Works | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Gene therapy for ALS: A review: Molecular Therapy
Frontiers | The Need for SMN-Independent Treatments of Spinal Muscular Atrophy (SMA) to Complement SMN-Enhancing Drugs | Neurology
Gene therapy - Wikipedia
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text
How It Works | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
What is SMA? | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Advances in Treatment of Spinal Muscular Atrophy – New Phenotypes, New Challenges, New Implications for Care - IOS Press
MOA | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors - ScienceDirect
