Teenage brothers Lucas (16) and Zane (14) Braun from Wingham are undergoing clinical drug trials to help slow down the progression of Morquio A Syndrome. It’s a rare genetic disorder that will cut their lives short by decades.
The brothers are among the first in Canada to receive the new treatment and they say it’s improving their quality of life. They want more people living with Morquio A to get the same treatment.
They’re on a drug called Vimizim. This week the makers of the drug applied to have it approved by Health Canada. Clinical trials show the drug has improved mobility, endurance, energy levels of test patients.
“It has also decreased the amount of sugars in the body and hopefully will slow down the progression of the disease.” Metabolic Geneticist Doctor Julian Raiman says.
The pair is missing an enzyme that breaks down complex sugars causing dwarfism, respiratory problems and extreme exhaustion. Roughly 3,000 people worldwide are living with Morquio A Syndrome.
“We know how each other feels. If I feel pain right now, Luke would feel the same thing.” Zane says.
There are few treatments available for the condition but Vimizim has given Debbie Braun hope that her boys will have a longer life.
“This drug therapy has actually given us hope,” She says. “It’s that hope they took away from me 14 years ago. They’ve kind of given it back.”
Published Sunday, December 8, 2013 5:37PM EST
Last Updated Sunday, December 8, 2013 7:09PM EST