Terry Pirovolakis receives numerous messages weekly from strangers seeking help for rare diseases affecting their children. The requests come from across the globe, detailing heartbreaking stories of genetic conditions causing muscles to weaken, neurons to misfire, and eyes to fail. One such plea came from Rebekah Lockard in Littleton, whose daughter Naomi was diagnosed with hereditary spastic paraplegia-50, a condition that impacts only around 100 individuals worldwide. These messages highlight the complexities of rare diseases and the desperate search for treatments. To learn more, continue reading the full story on STAT+.
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STAT+: This father built a gene therapy for his son. Now comes the harder part: saving others’ children, too
