Methylmalonic acidemia due to methylmalonyl-CoA epimerase deficiency Advocacy Hub | UniteRare

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Regulatory Watchboard

2 events

Mar 2022An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705

ModernaTX, Inc. — PHASE1, PHASE2

TrialRECRUITING

Aug 2021A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

ModernaTX, Inc. — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

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FINANCIAL LANDSCAPE SUMMARY

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2 active trials

2PHASE1, PHASE2

2Total recruiting

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Recent News & Research

1 article

Mol Ther Nucleic AcidsJun 16, 2026

The post-pandemic pivot: mRNA therapeutics enter the chronic rare disease arena

Published in Mol Ther Nucleic Acids. Giangrande PH et al.

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