Local family tries to raise awareness about rare diseases

Now four years old, Armand Hayes began life as a happy and seemingly healthy young boy with a voracious appetite.

“He used to eat man-sized meals; he would even take food off of my plate,” said his father, Tim Hayes of Miamisburg.

That was before Armand was diagnosed with GM1 gangliosidosis — a so-called rare or orphan disease that affects fewer than 200,000 Americans.

Today, Armand is confined to a wheel chair, eats through a feeding tube and takes medication to control seizures resulting from the crippling genetic disorder that progressively slows cognitive and muscular development and is likely to end in death if a cure is not found.

“He was diagnosed just before his second birthday,” the father said. “He was developing normally for the first couple of years of his life, but had some trouble walking. He was able to get where he was going, but at 18 months he was still stumbling.”

Armand’s doctors initially thought he might have muscular dystrophy, but blood tests were inconclusive. It took a series of DNA tests, muscle biopsies and MRIs before doctors at Cincinnati Children’s Hospital Medical Center came up with the answer in early 2011.

For Armand and his family, who are doing the best they can to manage his disease with available treatments, the news was bitter-sweet. They finally knew what the problem was, but they also discovered there was no cure.

They weren’t alone.

There are over 7,000 rare diseases affecting approximately 30 million Americans, yet fewer than 5 percent of those disorders have therapies approved by the U.S. Food and Drug Administration, according to the National Institutes of Health.

“It’s not just Armand,” said his grandmother, Judy Hayes. “In cities nationwide, there are millions of patients just like Armand. We want to raise awareness for all of these kids. We have to do this because nobody else will.”

Tim Hayes and his mother appeared before Montgomery County Commissioners last month to help convince city officials that rare diseases deserve to be addressed as a public health issue.

Following his presentation, county commissioners issued a proclamation in recognition of Rare Disease Day, which was first launched in 2008 and is observed internationally on the last day of February.

Tim Hayes hopes his efforts, including regular updates about Armand on his blog Armand’s Hope — www.armandshope.com — will inspire drug companies and medical researchers to make an extra effort to come up with new, innovative therapies for orphan diseases — even if it’s not in their best interest financially. And he hopes to provide a network for sharing new discoveries and treatments with other families struggling with rare diseases.

“One of our major goals is to not only spread awareness to healthy people, but also within the rare disease community,” the father said. “When Armand was diagnosed, we had no idea where to go from there, no support, no one who could understand what we were, and are, going through.

“Through the blog I keep, we’ve been able to meet with many families, and even get some of them into the same treatment Armand is in,” he said. “We’re trying to let other patients and families know that there is a large community out there for support, even within each disease niche.”

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