Spinal muscular atrophy: clinical trials to watch out for

Spinal muscular atrophy (SMA) is a rare genetic condition in which neurons in the spinal cord degenerate, leading to the loss of muscle tissue. This atrophy of muscle tissue causes those with SMA to suffer loss of muscular strength and movement. The neuromuscular condition is categorized into types 1, 2, 3 and 4, representative of the impact on motor function, which typically correlates with the age of onset. SMA is caused by mutation of the SMN1 gene in chromosome 5, which, when recessively inherited, causes a lack of functional SMN protein needed for motor neuron function. Multiple clinical trials are...