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Onasemnogene Abeparvovec: First Global Approval
Drugs, 2019 Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver a functional copy of the human survival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy (SMA).
Sheridan M. Hoy (4897468)
openaire +3 more sources
Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program [PDF]
Pediatric Neurology, 2022 BACKGROUND: Spinal muscular atrophy is a rare, neurodegenerative disorder caused by biallelic deletions in the survival motor neuron (SMN1) gene. Onasemnogene abeparvovec is a one-time, intravenous gene replacement therapy designed to deliver the SMN1 ...
Deepa Chand, Sandra P Reyna
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Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys [PDF]
Human Gene Therapy, 2022 Intravenous onasemnogene abeparvovec is approved for the treatment of spinal muscular atrophy in children
Mansfield, Keith +7 more
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Journal of Clinical Medicine, 2021 Background: Spinal muscular atrophy (SMA) is a neuromuscular progressive disease, characterized by decreased amounts of survival motor neuron (SMN) protein, due to an autosomal recessive genetic defect.
Corina Sporea +2 more
exaly +2 more sources
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Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial [PDF]
Neuromuscular Disorders, 2023 Bulbar function in spinal muscular atrophy has been defined as the ability to meet nutritional needs by mouth while maintaining airway protection and communicate verbally. The effects of disease-modifying treatment on bulbar function are not clear.
Sandra P Reyna
exaly +2 more sources