Gene replacement therapy in spinal muscular atrophy: filling the data gaps
Tim Hagenacker,
Ulrike Schara-Schmidt
Affiliations
Tim Hagenacker
Department of Neurology, Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Hufelandstr. 55, Essen 45147, Germany; Corresponding author.
Ulrike Schara-Schmidt
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
