Scientists in floppy baby breakthrough
Scientists believe they have made a breakthrough in the treatment of a muscle disease that leaves children severely paralysed.
Most children with floppy baby syndrome die before the age of one, but Australian researchers believe they may have found a way to help.
They were able to cure affected mice by replacing a missing muscle protein.
The team focused on proteins called actins. A gene called ACTA1 controls the production of actin in skeletal muscles.
It helps muscles to contract, but children with this disease have flawed versions of the gene and so the protein is not produced.
Dr Kristen Nowak, of the Western Australian Institute for Medical Research, who led the study, 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.”
