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Sangamo’s first gene-editing trial doesn’t convince investors

Sangamo’s first gene-editing trial doesn’t convince investors

these changes by conducting a controlled withdrawal of…ERT in patients enrolled in the study soon.”. ... Withdrawal from weekly ERT infusions would be “a very meaningful outcome” for patients with MPS II, he added.

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  • Making good practice common practice in rare disease

    symptoms. Literature suggests fatigue may be the first symptom to significantly improve with enzyme replacement therapy (ERT), a key treatment for Gaucher Disease, but this critical factor may be overlooked when

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    ERT is passionate and committed about transforming the way you conduct your drug development and healthcare efforts.

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