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

Inclusion Criteria

• •Patients are eligible to be included in the study only if all of the following criteria apply:
• •Confirmed diagnosis of isolated MMA due to MUT deficiency based on the following criteria:
• •* Elevated plasma methylmalonic acid concentrations (≥ 100 µmol/L)
• •* Presence of normal serum/plasma Vitamin B12 and plasma homocysteine levels
• •* Confirmed diagnosis by molecular genetic testing
• •Patient must be ≥ 1 year of age at the time of consent/assent (Inclusion of the first three patients will be restricted to individuals age ≥ 8 years)