Therapeutic interventions for spinal muscular atrophy: preclinical and early clinical development opportunities.
Laurent ServaisGiovanni BaranelloMariacristina ScotoAurore DaronMaryam OskouiPublished in: Expert opinion on investigational drugs (2021)
Children treated after the onset of symptoms continue to have significant disability. Given the heterogeneity of the population phenotype evidenced by variable response to initial therapy, age at treatment onset and the need to demonstrate added value beyond approved therapeutics, the clinical development of new drugs will be challenging.