Leukodystrophy

Why we need to “Find a Cure for VWM”?

Imagine for a moment that a child you love, perhaps your son,daughter, granddaughter, or niece, was unexpectedly diagnosed with one of those “parent’s worst nightmare diseases.” In this instance, the monster disease is Leukodystrophy called Vanishing White Matter disease.

Vanishing White Matter disease (VWM), also known as Childhood Ataxia with Central Nervous System Hypomyelination (CACH), is a devastating condition that destroys myelin” the brain's white matter. Unfortunately, there is no known cure or form of treatment for this disease. Symptoms generally appear in young children who appear to be developing fairly normally. A diagnosis is usually made in the early years of life at around 2 to 6 years old. According to the United Leukodystrophy Foundation, www.ulf.org - http://ulf.org/what-is-leukodystrophy VWM/CACH is one of 40 known forms of leukodystrophy.

Like many of you, I was once ignorant about VWM. I never heard of the disease and did the thought of it ever affecting my life never even crossed my mind. But then in 2009, my niece was diagnosed with VWM Leukodystrophy, and ever since that moment I’ve been searching the web like crazy, day and night, trying to help my sister save her daughter. I can still remember clearly the day my sister told me about the MRI results, and how they showed signs of Leukodystrophy. I was devastated, and the more I read about Leukodystrophy the more horrified I would get. My heart sank when in one article I read stated, “These disorders are progressive, meaning that they tend to get worse throughout the life of the patient”. All Leukodystrophies are a result of problems with the growth or maintenance of the myelin sheath. However, there are many genes that are important in this process. Leukodystrophies also share some common characteristics with Multiple Sclerosis (MS). The symptoms of the individual Leukodystrophies may vary because of the differences in their genetic...