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Gene therapies for rare diseases are under threat. Scientists hope to save them.

www.nature.com Gene therapies for rare diseases are under threat. Scientists hope to save them

As industry steps aside, scientists seek innovative ways to make sure expensive treatments can reach people who need them.

Gene therapies for rare diseases are under threat. Scientists hope to save them

"We have the technology, but we’re not going to save your kid because we can’t make any money on you’? Really?”

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