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Molecular and Cellular Pediatrics
Published in: Molecular and Cellular Pediatrics 1/2023

Open Access 01-12-2023 | Spinal Muscular Atrophy | Review

Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective

Authors: Michelle A. Farrar, Loudella Calotes-Castillo, Ranil De Silva, Peter Barclay, Lani Attwood, Julie Cini, Monica Ferrie, Didu S. Kariyawasam

Published in: Molecular and Cellular Pediatrics | Issue 1/2023

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Abstract

Onasemnogene abeparvovec has been life-changing for children with spinal muscular atrophy (SMA), signifying the potential and progress occurring in gene- and cell-based therapies for rare genetic diseases. Hence, it is important that clinicians gain knowledge and understanding in gene therapy-based treatment strategies for SMA. In this review, we describe the development and translation of onasemnogene abeparvovec from clinical trials to healthcare practice and share knowledge on the facilitators and barriers to implementation. Rapid and accurate SMA diagnosis, awareness, and education to safely deliver gene therapy to eligible patients and access to expertise in multidisciplinary management for neuromuscular disorders are crucial for health system readiness. Early engagement and intersectoral collaboration are required to surmount complex logistical processes and develop policy, governance, and accountability. The collection and utilisation of real-world evidence are also an important part of clinical stewardship, informing ongoing improvements to care delivery and access. Additionally, a research-enabled clinical ecosystem can expand scientific knowledge and discovery to optimise future therapies and magnify health impacts. Important ethical, equity, economic, and sustainability issues are evident, for which we must connect globally.
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Metadata
Title
Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective
Authors
Michelle A. Farrar
Loudella Calotes-Castillo
Ranil De Silva
Peter Barclay
Lani Attwood
Julie Cini
Monica Ferrie
Didu S. Kariyawasam
Publication date
01-12-2023
Publisher
Springer International Publishing
Published in
Molecular and Cellular Pediatrics / Issue 1/2023
Electronic ISSN: 2194-7791
DOI
https://doi.org/10.1186/s40348-023-00171-5

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