Spinal Muscular Atrophy: A modifiable disease emerges

Respiratory muscle failure and death in infancy or early childhood were the hallmarks of the heterogeneous neuromuscular disorder Spinal Muscular Atrophy [SMA] in the absence of intervention with mechanical ventilation and subsequently with the portable and more practical non-invasive respiratory support [NIV] [1]. Many paediatric respiratory centres around the world favoured supportive care without progression to invasive ventilation in the absence of effective therapies for SMA. However, others argued for the use of invasive ventilation for children regardless of the availability of effective treatments [2].

Source: Paediatric Respiratory Reviews - July 12, 2018 Category: Respiratory Medicine Authors: Dominic A. Fitzgerald, Francois Abel, Kristi J. Jones, Michelle A. Farrar Tags: Editorial Source Type: research