Mar 17 2016

Leukodystrophy Care Network Clinical Issues Survey

in news

Imagine your child experiencing an unexplained sudden loss of basic functions – a toddler who once constantly walked and ran, suddenly stumbling with each step; an infant with a healthy appetite, who once smiled and cooed, now crying inconsolably for every waking hour and only eating a few ounces of milk a day; a high school cheerleader suddenly losing all muscular control at a football game and shaking violently on the field. Each of these real-life examples is the result of a disease group called the Leukodystrophies.

Leukodystrophies makes up an inherited group of over 40 disorders of the white matter in the brain, affecting 1 in 7,000 individuals. Onset of symptoms can occur in the first months of life through adulthood, and can cause a rapid loss of abilities, such as voluntary movement and speaking, and result in severe morbidity and death. In spite of the thousands affected by these diseases, Leukodystrophies remain virtually unknown, even in the medical community. This often results in misdiagnosis and insufficient therapies and treatment options once a correct diagnosis is reached. Although currently there is no cure for any of the Leukodystrophies, they are all treatable. And, while all of them are terminal disorders, individuals affected by Leukodystrophies require and deserve expert medical care, essential to providing life-altering treatment and therapies to vastly improve health and overall quality of life.

Leukodystrophy Care Network’s (LCN) vision was launched in 2015 to:

“Establish a world-renowned LCN to exist across the United States, Canada, and eventually the world, to provide innovative therapies, treatment options, expert care and information to families affected by these diseases. Centers will be self-sustaining, yet collaborative, networked together to ensure that the highest quality groundbreaking care is available for all Leukodystrophy patients today and generations to come.”

The LCN is working with patients, their families, and clinicians to create “Care Guidelines,” so that children do not have to needlessly suffer from preventable and common disease-related issues.

If you or your family member is affected by a Leukodystrophy, please complete the Clinical Issues Survey to help us identify the most common issues that need to be proactively addressed.  Your participation will help the LCN develop multidisciplinary care guidelines to ensure all individuals affected by Leukodystrophies have access to the best quality of care possible.