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3 years ago

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The next generation of gene therapy for rare diseases forges ahead as developers weather hurdles

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The next generation of gene therapy for rare diseases forges ahead as developers weather hurdles

Even amid setbacks at the FDA, biopharma companies have been improving gene therapies to treat rare diseases like PKU, Fabry and gangliosidosis, with advances that range from new gene-insertion methods to technologies that can get the treatments to hard-to-reach areas of the body.

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health natural remedy fda public health public safety united states usa gene rare diseases science biology genetics

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The next generation of gene therapy for rare diseases forges ahead as developers weather hurdles

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The next generation of gene therapy for rare diseases forges ahead as developers weather hurdles

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