One in every thousand children has abnormalities of the white matter of the brain. Many of these disorders are genetically determined and progressive, associated with a fatal outcome. Paediatric neurologist Marjo van der Knaap has been investigating childhood white matter disorders for the past 25 years and has achieved some important breakthroughs that clear the way for an effective therapy. She hopes to see the search for a cure to be successful in her lifetime.
“Of course I’m upset every time a child dies. It breaks my heart to have to tell parents that their child has a leukodystrophy, and only has a short life ahead. But my job is to support them in their hour of need, to explain how we are going to deal with each and every problem. When I do that, I make the situation bearable for the parents. If I give way to my emotions, I cannot perform this role properly.” Marjo van der Knaap (born 1958) is professor of Paediatric Neurology at the VU Medical Centre in Amsterdam, where she performs research on white matter disorders in children. Leukodystrophies have catastrophic consequences, but Prof. Van der Knaap sees this as a challenge: “I’m not the kind of person you can get to do fundamental research on the common cold. Leukodystrophies are a terrible problem. We have to find a solution for them.”