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1d
Gene therapy may be 'one-shot stop' for rare bone disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
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BioSpace
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Opinion
Opinion: Companies Vie to Develop a Hunter Syndrome Therapy That Reaches the Brain
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...
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