# Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies Guidance for Industry:  Guidance for Industry - FDA Guidance Document

Source: https://www.globalkeysolutions.net/guidances/guidance-document/slowly-progressive-low-prevalence-rare-diseases-with-substrate-deposition-that-results-from-single-enzyme-defects-providing-evidence-of-effectiveness-for-replacement-or-corrective-therapies-guidance-for-industry-guidance-for-industry/edfb93d1-715f-42f9-b789-3cd598a3290d

> FDA guidance document: Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies Guidance for Industry:  Guidance for Industry. Issue date: July 27, 2018. Get complete insights and analysis.

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## Details

- Title: Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies Guidance for Industry:  Guidance for Industry
- Communication Type: Guidance Document
- Product Type: biologics
- Office Name: Center for Drug Evaluation and Research
- Office URL: /offices/center-for-drug-evaluation-and-research/cd980d74-1cbf-4226-ba9a-9045324c236c
- Issue Date: 2018-07-27
- Last Changed: 2024-04-11
- Docket Number: <a href="https://www.regulations.gov/docket/FDA-2018-D-2456">FDA-2018-D-2456</a>

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