NEW YORK (Reuters) - An experimental therapy for a rare, often fatal genetic disorder appears to offer hope for infants and very young children with the condition, according to data from a small clinical trial reported in the New England Journal of Medicine on Wednesday.

The enzyme-replacement drug, asfotase alfa, acquired by Alexion Pharmaceuticals Inc with its $610 million purchase of Canada-based Enobia Pharma, could become the first approved treatment for the metabolic disease hypophosphatasia, or HPP.

The condition is a genetic enzyme deficiency that causes bone softening and muscle weakness and can lead to severe lung problems and damage to other vital organs. It affects about 1 in 100,000 newborns worldwide, according to the National Institutes of Health. About half of infants with a severe form of the disease do not survive beyond one year.

In the study of 11 babies and toddlers under three with life-threatening HPP, treatment with asfotase alfa resulted in "striking" improvements in skeletal problems and dramatic improvements in lung function and mobility, researchers reported.

"I'm thrilled to see babies who were really doomed responding to the treatment," Dr Michael Whyte, the study's lead investigator, said in a telephone interview.

"The therapy is proving not only life-saving but also health-restoring," added Whyte, of Shriners Hospitals for Children in St. Louis, who has been working on this ultra-rare disease for more than 30 years.

Videos accompanying the online version of the study in the New England Journal of Medicine show dramatic motor skill improvements of two of the trial subjects. In one, a three-year-old who was unable to stand prior to therapy is shown climbing the steps of a small plastic slide after 24 weeks of treatment.

Respiratory function improved in all patients, researchers said. Ten of the 11 needed breathing support before treatment. After 48 weeks of treatment, only three of the nine children who remained on therapy needed help breathing.

Breathing problems arise as the shape of the chest becomes deformed due to soft bones in the thorax, which compromises lung function, Whyte explained.

'SHE CAN STAND ON HER OWN'

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Alexion drug offers hope for rare, deadly disorder

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