CNBC·10 min read

Rare disease is taking his sight. Access to a treatment is hard to get

G
Gerry Smith
Rare disease is taking his sight. Access to a treatment is hard to get
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CNBC Cures is proudly underwritten by Alexion, AstraZeneca Rare Disease, whose support enables our journalism to elevate stories that advance research, innovation and patient care in rare diseases.

If any parents should be able to help their sick child, it's Judy and Courtney Stecker.

They've both worked for the Trump administration, giving them high-level connections in Washington and at the nation's top hospitals. Doctors diagnosed their son, Wheeler, with a rare brain disorder as an infant, early enough to slow its progress. Scientists have developed a drug that could help him.

And yet, seven years after Wheeler was born with a form of Batten disease, he still hasn't received that treatment. And time is running out. Wheeler is going blind.

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