The Floppy Baby Syndrome

By Staff

Tuesday, June 9, 2009, 13:54

A breakthrough in the medical field has come as a boon for thousands of families across the globe. The new treatment will restore a muscle function known as Floppy Baby Syndrome. It is a congenital myopathy disorder that causes babies to be born without the ability to properly use their muscles.

The currently incurable genetic diseases render most of the affected children severely paralysed and take the lives of the majority of these children before the age of one. This new treatment is an important step towards one day hopefully being able to better the lives of human patients.

A team at the Western Australian Institute for Medical Research (WAIMR) has restored muscle function in mice with one type of Floppy Baby Syndrome. The mice with Floppy Baby Syndrome were only expected to live for about nine days, but they managed to cure them and thus allowing them to live naturally and very actively into old age. Mice who were cured of the disease lived more than two years, which is very old age for a mouse.

Dr Kristen Nowak, lead author on the publication, said the team was extremely encouraged that it had been able to cure a group of mice born with the condition. Dr Nowak said the team was able to cure the mice with the recessive form of the genetic condition by replacing missing skeletal muscle actin – a protein integral in allowing muscles to contract – with similar actin found in the heart.

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