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A Rare Disease and My Children’s Right to Try

March 4, 2024

FDA red tape kept effective treatment an ocean away.

While reading Judy Stecker’s “The FDA Could Help Save My Son From a Rare Disease” (op-ed, Feb. 28), I couldn’t stop thinking about my own children’s battle against a rare disease—and the Food and Drug Administration red tape that kept effective treatment an ocean away.

In 2020 my 1-year-old daughter, Olivia, was diagnosed with a rare and fatal genetic brain disorder called metachromatic leukodystrophy, which slowly robs patients of their motor functions. Three months later, our youngest daughter, Keira, then an infant, got the same diagnosis.

Devastatingly, it was too late to save Olivia—she’s now in hospice care. But since Keira was a newborn, there was a cutting-edge, personalized gene therapy that could help her by modifying her DNA.

Read the rest of the story at Wall Street Journal.

Kendra Riley is a business owner and mom of three girls, two of which were diagnosed with MLD in 2020. She has since become an advocate for other rare disease families like hers across the nation.



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