eP438: Ethical issues related to gene therapy with onasemnogene abeparvovec for spinal muscular atrophy type 1 in a developing country

The 5-q linked spinal muscular atrophy (SMA) (OMIM#253300) or SMA type 1 is an autosomal recessive, neuromuscular degenerative disorder caused by deletion or variants of SMN1 gene. SMA type 1 had been the leading genetic cause of infant mortality and gene replacement therapy with onasemnogene abeparvovec (AVXS-101, Zolgensma) was approved by the Food and Drug Administration (FDA) in May 2019. We described our experience of the first four Malaysian patients with SMA type 1 who received compassionate treatment in University Malaya Medical Center (UMMC), Malaysia and explored the main ethical issue related to this therapy.