A paper describing preclinical work that was foundational for the gene therapy for spinal muscular atrophy now sold as Zolgensma has been retracted for data inaccuracies. The article, “Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN,” was published in Nature Biotechnology in 2010. Its corresponding author, … Continue reading Paper by gene therapy Zolgensma developer retracted because of discrepancies in mouse survival rates