Randomisation versus prioritisation in a managed access programme: lessons from spinal muscular atrophy

Over the last few years, spinal muscular atrophy (SMA) has transitioned from being an untreatable, inexorably progressive disease to a condition for which there is now an approved therapeutic option, Nusinersen. Two additional therapeutic options are likely to become available in the very near future in Europe. Published [1] and emerging data from treated, pre-symptomatic patients indicate that a large majority of these individuals have, so far, a normal motor development. This has resulted in a resurgence of the efforts to initiate newborn screening programmes [2].
Source: Neuromuscular Disorders - Category: Neurology Authors: Tags: Editorial Source Type: research