New drug to help spinal muscular atrophy patients?RSS Feed

The spinal injury seen in spinal muscular atrophy (SMA) may not exclusively affect the motor neurones in the spinal cord as was previously thought, research has uncovered.

A study, published in journal Nature, revealed that defects in peripheral tissues such as liver, muscle and heart, may also contribute in severe cases of the disease.

The experiments were testing a new candidate drug to treat SMA in mice.

It was found to significantly suppress symptoms when injected under the animals' skin.

Cold Spring Harbor Laboratory's Professor Adrian Krainer commented: "These systemic, or subcutaneous, injections, extended the lifespan of mice that have the equivalent of severe human SMA by 25-fold."

There is no current cure for spinal muscular atrophy, with treatments focusing on relieving symptoms and providing support and care.

These treatments include breathing exercises, physiotherapy, back braces, surgery, diet and exercise.

The disease affects the sufferer's physical abilities, such as moving, walking and breathing, but has no affect on their mental development.

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Posted by Paul Breen
 

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