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2024 update: European consensus statement on gene therapy for spinal muscular atrophy

12 Jun 2024

Abstract excerpt

Spinal muscular atrophy (SMA) is one of the most common genetic diseases and was, until recently, a leading genetic cause of infant mortality. Three disease-modifying treatments have dramatically changed the disease trajectories and outcome for severely affected infants (SMA type 1), especially when initiated in the presymptomatic phase. One of these treatments is the adeno-associated viral vector 9 (AAV9) based...

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PMID: 38878702
DOI: 10.1016/j.ejpn.2024.06.001

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