Scientists have made a breakthrough In the treatment of a muscle disease called floppy baby syndrome.
Babies born with this disorder are severely paralyzed and most of them die before the age of one. The study was done on affected mice. The Australian team which carried out the study were able to cure affected mice by replacing a missing muscle protein.
T the findings, in the Journal of Cell Biology, could also lead to improved movement for affected babies.
Actins is the team that focused on proteins. ACTA1 is a gene which controls the production of actin in skeletal muscles. Children with this disease have flawed versions of the gene and thus the protein is not produced.
It has also been observed by scientists that children with floppy baby syndrome were not totally paralysed at birth.
Dr Kristen Nowak, of the Western Australian Institute for Medical Research, led the study. He said: “The mice with floppy baby syndrome were only expected to live for about nine days, but we managed to cure them so they were born with normal muscle function, allowing them to live naturally and very actively into old age.
“This is an important step towards one day hopefully being able to better the lives of human patients – mice who were cured of the disease lived more than two years, which is very old age for a mouse.”
Professor Nigel Laing, head of the research group, said: “Current therapies only target the effects of these conditions, not the condition itself – we hope our approach could lead to a much greater improvement for a range of muscle diseases.”
The development of their gross motor skills such as sitting and crawling is delayed causing floppy baby syndrome. It is a gene related disorder.
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