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Medicaid Is A Lifeline For Children With Rare Diseases

As America’s lawmakers debate various ways to fix our broken health care system, we in the rare disease community are alarmed by proposed cuts to Medicaid funding. The term “rare disease” is a bit paradoxical. When viewed individually, a particular disease may affect a minuscule portion of the population. But when considered as a whole, roughly 1 in 10 Americans live with a rare disorder. For some the rare disease is progressive and eventually fatal, as in the case of those of us parents who have watched our children develop typically before the onset of a neurodegenerative disease slowly robs them of their function and eventually their lives. For others, the disorder is manageable with diet modifications, scrupulous monitoring by medical specialists, or changes to our environments that allow us to work and function in society.

Nearly all of us have lived through the frustrating experience of the “diagnostic odyssey.” The diagnostic odyssey is a term used to describe the journey through the medical system a rare disease patient makes in order to receive a diagnosis. The average rare disease patient often waits months to years for a diagnosis and is misdiagnosed multiple times. The financial devastation for a patient on this journey is often extraordinary and the prolonged time spent searching for a diagnosis has a negative impact on an individual’s ability to stay employed and maintain insurance coverage. At the end of this odyssey, the relief of finally getting an answer is quickly replaced by the painful fact that only about 300 of the 7,000 rare diseases have any sort of an effective treatment.


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