As part of our ongoing aim to bring you reports of significant or innovative developments, we have added a review of the SPR1NT trial which investigated the efficacy and safety of onasemnogene abeparvovec in pre-symptomatic infants at risk of spinal muscular atrophy.
Independent commentary is provided by Dr Sandi Kariyawasam, a Paediatric Neurologist at Sydney Children’s Hospital, Randwick and Associate Lecturer at the School of Women’s and Children’s Health, University of New South Wales.
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