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Clinical and Laboratory Study of Methylmalonic Acidemia

for
Organic Acidemia, Methylmalonic Acidemia,
Location: Bethesda MD, Pittsburgh PA
Sponsor: National Human Genome Research Institute (NHGRI)

Sex: All

Age: 0 - 70+

Code: NCT00078078

Recruiting

An 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

for
Methylmalonic Acidemia
Location: Toronto, Edmonton, San Diego CA, Los Angeles CA,
Sponsor: ModernaTX, Inc.

Sex: All

Age: 1+

Code: NCT05295433

Phase1, Phase2, Recruiting

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

for
Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia
Location: New York NY, Washington DC, Tampa FL, Indianapolis IN,
Sponsor: Recordati Rare Diseases

Sex: All

Age: 0+

Code: NCT05040178

Recruiting

Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study

for
Malonic Aciduria, Methylmalonic Acidemia
Location: Montreal
Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre

Sex: All

Age: 0+

Code: NCT01289158

Recruiting

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