Advocacy intelligence hub — real-time data for patient organizations
Astellas Gene Therapies — NA
Astellas Gene Therapies — PHASE1
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
Total programs
Pompe Disease Newborn Screening Shows 60% Faster Treatment
States implementing Pompe newborn screening reported 60% reduction in time to treatment initiation.
Newborn Screening Expands to Include More Rare Diseases
The Advisory Committee recommended adding Duchenne MD and Pompe disease to the Recommended Uniform Screening Panel.
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