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WASHINGTON — Jennifer Puck has successfully treated 10 children with a gene therapy for a fatal disorder that decimates their immune system. But she has no idea how to get her drug approved and frankly is running out of ideas.

“I wish I had a clue about where to go from here,” said Puck, an immunologist at University of California, San Francisco, from a plush chair above Union Station. 


The problem is simple: Size. Puck’s therapy is for a disease, Artemis-SCID, that affects just two to three new U.S. patients every year — far too few for a company to generate a profit, or to even run the kind of studies regulators usually demand before approving drugs.

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