Murrells Inlet family advocate for research on rare disease

MURRELLS INLET, SC (WMBF) - A Murrells Inlet family is back from a trip to Capitol Hill advocating to continue research funding for a rare disease called Tuberous Sclerosis Complex.

Heather Harden said her daughter Nell was only ten months old when she noticed white spots on her skin. She later found out those spots were much more when doctors diagnosed her with TSC.

TSC is a rare genetic disorder causing non-cancerous tumors to grow on different organs.

Harden said Nell looks and acts like the typical nine-year-old, but she has tumors on her brain, heart, kidney, skin and eyes.

TSC can also lead to even more issues, including seizures, kidney disease and learning delays.

Harden and Nell met with six South Carolina congressmen, including Russell Fry and Jeff Duncan, encouraging them to write dear colleague letters and push other lawmakers to continue government funding for the TSC research program.

Harden said the research program has produced the only three drugs that help treat TSC.

So, she said it’s important research funding is renewed each year.

She said research has led to huge strides for her daughter Nell, significantly shrinking her heart and skin tumors.

“It works,” Harden said. “So, without that initial research, we would never have these drugs. I can’t imagine where we would be otherwise without this program.”

Nell said she only has mild symptoms, so it’s important to use her voice and advocate for those who can’t make the trip to D.C.

“If we get donated money, then we can try to find a cure,” Nell said. “People that have TSC, most of them are way worse than I am or probably will be.”

The government has provided more than $100 million towards the TSCRP over the last two decades.

The Hardens said making the trip ensures the rare disease does not slip through the cracks.

Copyright 2024 WMBF. All rights reserved.