Time Running Out For Children With Rare Disorder

"The federal government defines a rare disease or condition as one that affects fewer than 200,000 Americans. What then do you call a disease that affects just 200 people?

"One of the rarest of the rares," said Cate Walsh Vockley of the National Niemann-Pick Disease Foundation.

A family with strong local ties found out the hard way. After countless doctor visits, tests and misdiagnoses, Salem native Bryan Hadley and his wife, Laura, were told their 10-year-old son, Peyton, has the genetic metabolic disorder.

Niemann-Pick Type C usually strikes children of school age. It does so by interfering with their ability to metabolize cholesterol, causing harmful amounts to accumulate in the spleen, liver and brain, and leading to neurological deterioration.

It is fatal.

The news was unbearable for the couple, who live in Medford and have three kids. They had passed on a disease for which there is no cure and very few treatment options.

The Hadleys fear their daughter, Kayla, 7, also has it and are awaiting DNA test results. (There is a 25 percent chance with each pregnancy.)"


The Hadleys have their own website, Hadley Hope. It's an informative website about Niemann-Pick Type C disease. Their children (those test positive) will be on a new medication (called Zavesca), which is supposed to be "the most promising drug available to slow the progression of the disease, and improve eye movement, swallowing, and other symptoms". The insane part is how much the medication costs: over $9000 per month for one child. (And their insurance has only promised to cover 50%, for a 1- yr period).

It's entirely unfair that these adorable children are/could be facing such a short future- and that these parents have to face such outrageous medical costs! But I did notice on their website that they ask people who have children who might come into contact with their kids, to keep the information "confidential". If their friends don't know they're sick, they won't be ostracized or treated differently- thus, they will hopefully have the quality of life they deserve, for the remainder of their lives.