Federal funding fight hits home for families afflicted by rare diseases

Editor's note: Este artículo está traducido al español.

The warning signs emerged within weeks of birth — jaundice that wouldn’t fade, mysterious liver problems and an enlarged spleen.

By 2 months old, Las Vegas newborn Liam Mitchell had received a diagnosis that would reshape his family’s entire world: Niemann-Pick disease type C (NPC), a devastating genetic disorder sometimes called “childhood Alzheimer’s.”

This rare condition prevents the body from properly processing and transporting fats like cholesterol into cells, leading to a cascade of neurological damage.

For families facing such rare diseases, hope hinges on medical research — but funding remains critically scarce. With NPC affecting an estimated 1 in 1 million Americans, research depends almost entirely on grants from the N