Nowadays, there are three drugs in research pipelines using this approach for ABCA4‐associated Stargardt diseases, dysferlinopathy associated with the DYSF gene, Usher syndrome type 2 B caused by mutations in MYO7A gene and hearing loss due to OTOF malfunction (Table S3). Here, DYSF is linked to neuromuscular disease caused by qualitative or quantitative defects of dysferlin.