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Friday, 28 July, 2000, 01:47 GMT 02:47 UK
Rabbit milk saves babies
Rabbit milk helped to treat a potentially fatal muscular disorder
Milk from genetically-modified rabbits has been successfully used to treat four babies suffering from a potentially fatal disease.

Pompe's disease is a muscular disorder caused by deficiency of an enzyme called a-glucosidase.

Children who suffer from the disease deteriorate rapidly, and usually die before they reach their first birthday.

They suffer from breathing and feeding difficulties, a lack of muscle strength and disease of the heart muscle.

For the first time, there is a treatment for Pompe's disease with clearly beneficial effects

Dr Ans van der Ploeg, Sophia Children's Hospital, Rotterdam

Often they are unable to perform even the most basic functions, such as rolling over, sitting and standing.

Researchers from Sophia Childrens Hospital, Rotterdam, The Netherlands, treated four babies with Pompe's disease with a form of the enzyme derived from rabbit's milk.

The rabbits had been given a human gene which caused them to produce milk containing the enzyme.

Stimulated muscle

The enzyme stimulated the babies' muscle to function properly, built up strength and led to improved heart function.

One of the babies, who had the least severe form of the disease, was able to stand up with support by the age of 12 months - something that is usually totally beyond a child with Pompe's disease.

The two babies with the most severe form of the disease had lost the ability to use their muscles almost completely before they were given treatment.

During treatment, both became dependant on a respirator to breathe.

However, by the end of their course both had become hospital outpatients, and had also showed progress in mental development.

The investigators recommend that treatment should be started early.

However, they warn that larger studies are needed to corroborate their findings, and to monitor the long-term impact.

Lead researcher Dr Ans van der Ploeg said: "For the first time, there is a treatment for Pompe's disease with clearly beneficial effects."

The researchers added: "The milk of transgenic animals seems to be a safe source and opens the way for further exploration of this production method."

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