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Start Over You searched for: Languages English Remove constraint Languages: English Publication Year 2020 Remove constraint Publication Year: <span class="single" data-blrl-single="2020">2020</span>

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518. Slowly progressive, low-prevalence rare diseases with substrate deposition that result from single enzyme defects: providing evidence of effectiveness for replacement or corrective therapies

520. Policy for certain REMS requirements during the COVID-19 public health emergency: guidance for industry and health care professionals