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A rare disease is taking his vision. A gene therapy could help — if he can get it

A well-connected family’s fight to access a potential treatment for their son highlights the barriers most rare disease patients struggle to overcome.

A rare disease is taking his vision. A gene therapy could help — if he can get it

Published September 10, 2026 · Category: Finance

Overview

A well-connected family’s fight to access a potential treatment for their son highlights the barriers most rare disease patients struggle to overcome.

Source

Originally published at www.cnbc.com.

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