Every parent wants the best for their child, particularly when it comes to their health. For families confronted by spinal muscular atrophy – a rare but debilitating genetic disorder – the list of symptoms makes for grim reading indeed.
According to the US Muscular Dystrophy Association, infants with SMA type 0 are often born severely weak. Such babies can have facial paralysis and congenital heart defects. Heartbreakingly, some patients can die from respiratory failure by six months of age, others within their first month. Older sufferers can have severe disability as motor neurons – nerve cells that carry signals from the brain to the muscles – are affected, causing weakness in the limbs and other problems, such as difficulty breathing or swallowing.
Compounding the cruelty of this illness is the cost of treating it. The best remedy for SMA is gene therapy but this cutting-edge medicine is beyond the reach of most. One drug, Zolgensma, has been described as the world’s most expensive medicine, costing about $2 million for each patient.
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