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The Floppy Baby Syndrome

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.
Disclaimer: The information provided in this article is for general informational and educational purposes only and is not intended as a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or a qualified healthcare provider with any questions you may have regarding a medical condition.



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