There are no approved medical treatments for hypophosphatasia, according to the study.
The current study involved 11 children. All were given an initial intravenous infusion of asfotase alfa, followed by shots of the medication three times a week.
Parents of one baby removed their child from the trial during the initial intravenous treatment. A second baby died from an unrelated infection after more than seven months of treatment.
The remaining nine children have received at least 18 months of treatment with asfotase alfa.
X-rays taken at the start of the study and at weeks 24 and 48 showed significant improvement in bone formation after treatment. In addition, the babies showed improvement in lung function, physical skills, and in the development of intelligence, according to the study.
The treatment was "very well tolerated," Whyte said. And, he added, there was no evidence that the children were developing resistance to the drug.
Treatment with asfotase alfa needs to be ongoing, and it's not yet clear if there are long-term side effects. Whyte and his colleagues are continuing to study the patients enrolled in this trial. He said that he believes children born with the severe or life-threatening form of the disease should be given this medication, even though it's still considered experimental. The reason, he said, is the severe form of this disease is "invariably lethal, usually soon after birth."
Dr. Spyros Mezitis, an endocrinologist at Lenox Hill Hospital in New York City, said the research is promising an
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