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Canadian drug for rare bone disease shows promise
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CTVNews.ca Staff
Date: Sat. Jan. 14 2012 10:17 PM ET
Clinical trials of a Canadian-made drug used to treat a rare brittle bone disease appear to be so promising that it could be approved within five years, half the time it usually takes to get a medication to market.
Hypophosphatasia robs bones of the minerals needed to keep them strong, causing them to be so weak that they are easily broken. In infants, a diaper change could lead to fractures. Older children can experience premature tooth loss (before age five), delayed walking, as well as frequent fractures.
But at the Health Science Centre in Winnipeg, ENB-0400, which is manufactured by Montreal-based Enobia Pharma, the clinical trial's results have been promising.
"In every single case we've seen improvement. Radiologically, clinically and functionally," Dr. Cheryl Greenberg, head of the Department of Pediatrics and Child Health, told CTV News.
Greenberg has been conducting clinical trials with the drug for four years in Winnipeg.
Dozens of children and adults from across Canada and around the world have travelled to the city's Health Sciences Centre for treatment, including 15-month old Gideon Siemens.
His parents, Matt and Nicole, took their son to Winnipeg a year ago. Gideon was born with hypophosphatasia, and at birth his ribs fractured and his legs broke.
"He was so brittle," Matt Siemens told CTV. "You had to move him so carefully."
Gideon receives injections of ENB-0400 three times a week, and now his bones grow and are getting stronger.
"It flat out works," Matt Siemens said. "The drug they have works. This is living testament to it here."
According to statistics on the drugmaker's website, one in 100,000 people is estimated to have the rare bone disease. And the highest known incidence is within Manitoba's Mennonite population, where one in every 2,500 newborns has the disease.
The side effects of the drug are limited and it could be up for approval within the next year or two.
"It really doesn't get any better than this," Greenberg says. "To take a baby who has a fatal disease and turn it into a treatable disease."
With a report from CTV's Jill Macyshon
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