Red tape, rare disease, and a polar vortex

CHARLIE AND KOLTON MARTIN (COURTESY ALYSSA MARTIN)

Menkes disease is a devastating disorder. A rare inherited condition affecting copper metabolism, it causes neurodegeneration and often kills affected boys by age 3. But there's hope that injecting Menkes boys with an experimental medication may help some of them — if they're treated early enough.

That's why clinicians believed it was so crucial to start early treatment for Charlie and Kolton Martin, two twin boys born in Iowa in December. But the process involved in obtaining the experimental drug thought to be their best shot turned out to be harder than anyone imagined.

STAT's Rebecca Robbins has the story of the Iowa clinicians who scrambled and overcame obstacle after obstacle — including last week's polar vortex that sent temperatures plummeting across the Midwest — to try to save the Martin boys.

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