With arrival of breakthrough therapies, a rare disease group reconsiders the possible — and its mission

DALLAS — Inside Kelly Eakin’s garage in suburban Maryland, two of the world’s smallest wheelchairs gather dust.

Just a few years ago, these wheelchairs were in high demand. There was a constant waiting list of eight or nine young children with spinal muscular atrophy, or SMA, a devastating disease that could shorten life and stunt growth so severely many patients couldn’t use the child wheelchairs insurance covered. So the Fighting for Kaiden Foundation, of which Eakin is president, ordered 7-pound ones from a Swedish manufacturer at $6,000 or $7,000 a pop. 

Since December 2016, though, three powerful SMA therapies have been approved. These drugs have dramatically altered the outlook for anyone born with the rare neuron-wasting disease, allowing many to not only live and grow but even walk. 

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