The Globe and Mail - "The Agonizing Fight for Isaac: the Hope and the Hurdles"
Andrew McFadyen lives with the agony of knowing that a ground-breaking treatment for his son's debilitating disease may be just out of reach. His son Isaac was born with MPS VI, an extremely rare metabolic disorder. At age 2, Isaac was featured in a Globe and Mail series that led to the Ontario government's decision to fund Naglazyme, the only available treatment for his disease, which costs an annual $300,000 to $1-million a patient. But researchers at The Mount Sinai Hospital have come up with an experimental treatment for MPS VI. In a study published January in the online journal PLoS One, rats with the disease showed remarkable improvement in mobility and other indicators after taking pentosan polysulfate, an anti-inflammatory drug that costs about $7 a pill. Learn more