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Results for “Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency”

4 trials

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Showing 4 of 4 results

Not applicableNot Yet RecruitingNCT07432880
What this trial is testing

A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene

Who this might be right for
Methylmalonic Acidemia (MMA)
Genespire Srl 30
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Testing effectiveness (Phase 2)WithdrawnNCT03810690
What this trial is testing

Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

Who this might be right for
Methylmalonic Acidemia (MMA)Metabolism, Inborn Errors
ModernaTX, Inc.
Testing effectiveness (Phase 2)Active Not RecruitingNCT04899310
What this trial is testing

Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

Who this might be right for
Methylmalonic Acidemia
ModernaTX, Inc. 74