As a mom to 2 (now adult) children with an ultra rare disease, this breaks my heart into pieces. There is also a potential therapy for our kids, but even growing their own stem cells in a lab is too cost prohibitive for our family (x2 kids)
Reposted from Chris Morran
Whenever drug companies justify their high prices, they tend to claim a need to recoup their huge investment in developing that drug.

The story of one drug that costs more than $2M for a single dose exposes the truth about who is paying to develop the world's most expensive medications:

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