It is inherited, with parents passing the genes to their children. Families often feel terribly isolated, she said. Like 4H leukodystrophy, they are often progressive and mainly affect children, and have limited or no treatment options.ĤH leukodystrophy is rare, but the burden on patients, families and the health-care system is enormous, said Bernard, who is recognized as a world expert in leukodystrophies. “Rare diseases are often overlooked because individually they are rare - but together they are not,” she said.Ĭollectively, rare diseases affect about three million people in Canada, including 500,000 in Quebec. September is Leukodystrophy Awareness Month, and Bernard sees it as “a very good opportunity to sensitize people to rare diseases.” “I think false hope is worse than no hope,” she said.
“The idea is to find a cure, so if we don’t have a good model, we can’t test treatments,” she said in an interview.īernard won’t put a timeline on when treatment could become available. It is considered a crucial step in the development of treatments, said Bernard, a scientist in the Child Health and Human Development Program at the RI-MUHC and a neurologist at the Montreal Children’s Hospital.
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