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BIO Comments on FDA Draft Guidance on Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition Resulting from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies

September 25, 2018

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition that Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies.

It’s important to support and advance drug development for individuals with rare diseases. BIO suggests the FDA eliminate the phrase “low-prevalence rare disease” from the guidance.

Download Full Comments Below
BIO Comment Letter Slowly Progressive, Low-Prevalence Rare Diseases FDA 2018-D-2456
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