Alerts
PDF
Other content recommended for you
- Gene therapy for neuromuscular disorders: prospects and ethics
- Spinal muscular atrophy: untangling the knot?
- A current approach to heart failure in Duchenne muscular dystrophy
- The transthyretin amyloidoses: advances in therapy
- Long-term treatment with leuprorelin for spinal and bulbar muscular atrophy
- Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen
- A 2020 centenary perspective on neuromuscular disorders
- HnRNPR strongly represses splicing of a critical exon associated with spinal muscular atrophy through binding to an exonic AU-rich element
- Duchenne muscular dystrophy: genome editing gives new hope for treatment
- Great expectations: virus-mediated gene therapy in neurological disorders