Gene therapy may help children born with 'bubble boy' disease
Ten children with an especially rare and hard-to-treat form of "bubble boy" disease are living normal lives after receiving a new gene therapy approach, researchers say.
Source: upi.com
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Ten children with an especially rare and hard-to-treat form of "bubble boy" disease are living normal lives after receiving a new gene therapy approach, researchers say.
As gene therapies for rare diseases find success, more cracks are appearing in the current regulatory and commercialization models.