8 April 2017 - Powerful new pharmaceuticals are promising to change the lives of those who suffer from rare diseases. But with some governments balking at covering the sky-high cost of those drugs, writes Kelly Grant, patients are insisting they shouldn’t be asked to keep suffering – or find a way to pay.

In many ways, Michael Luckevich is the lucky one in his family. The 58-year-old Seattle-based big-data analyst can climb the stairs of his home without worrying that his legs might collapse beneath him. With effort, he can haul himself out of a chair on his own. His body isn’t racked by severe chronic pain – although, until recently, at least, he often suffered low-grade discomfort that led him to put off physical tasks, like fixing the pipes under his sink, because he didn’t think his aching body could handle the strain.

His sisters, Theresa and Rosanne, on the other hand, aren’t so fortunate. Theresa, a 62-year-old former nurse, has the most serious case of the rare genetic disease that afflicts four of the eight siblings in their family: hypophosphatasia, an inherited disorder in which the skeleton fails to mineralise; in the worst cases, those involving babies, HPP leaves the bones soft as wet noodles.

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