Gene replacement therapy in spinal muscular atrophy: filling the data gaps

Affiliations

• ¹ Department of Neurology, Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Hufelandstr. 55, Essen 45147, Germany.
• ² Department of Pediatric Neurology, Center for Neuromuscular Disorders, Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Hufelandstr. 55, Essen 45147, Germany.