Mechanism of Action | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Base editing rescue of spinal muscular atrophy in cells and in mice | Science
The approved gene therapy drugs worldwide: from 1998 to 2019 - ScienceDirect
Mechanism of Action | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Frontiers | The Need for SMN-Independent Treatments of Spinal Muscular Atrophy (SMA) to Complement SMN-Enhancing Drugs
Frontiers | Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment
Bridging the Gap: Gene Therapy in a Patient With Spinal Muscular Atrophy Type 1 | Neurology
Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease
Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine - ScienceDirect
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders | Neurological Research and Practice | Full Text
Advances and limitations for the treatment of spinal muscular atrophy | BMC Pediatrics | Full Text
IJMS | Free Full-Text | Drug Discovery of Spinal Muscular Atrophy (SMA) from the Computational Perspective: A Comprehensive Review
Mechanism of Action | ZOLGENSMA® (onasemnogene abeparvovec-xioi)
Zolgensma for the Treatment of Spinal Muscular Atrophy, Novartis
Frontiers | History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy
Novartis' gene therapy Zolgensma gets Japan approval for neuromuscular disorder | S&P Global Market Intelligence
Gene therapy for ALS: A review: Molecular Therapy
Illustration of therapeutic approaches in SMA involving molecular... | Download Scientific Diagram
New Drug Product: Zolgensma - MPR
Mechanisms of action of main molecular therapeutic strategies that are... | Download Scientific Diagram
Gene therapy successes point to better therapies | PNAS
Comparative evaluation of AAV gene therapy, antisense therapy and small molecules therapy for treatment of SMA for efficacy and
