Rare disease is taking his sight. Access to a treatment is hard to get
The parents of a child suffering from a rare brain disorder called Batten disease are struggling to secure access to a potentially life-saving treatment. Despite their high-level connections, regulatory hurdles and clinical trial exclusions have left the family in a difficult position.
Why it matters
The story illustrates the systemic challenges patients face in accessing experimental treatments for rare diseases, even when they have significant resources.
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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