HemoShear - Model HST5040 - Investigational Oral Small Molecule Therapy for MMA and PA - Datasheet
Methylmalonic acidemia (MMA) and propionic acidemia (PA) are rare intoxication-type inborn errors of metabolism that are characterized by acute metabolic decompensations, acidosis and hyperammonemia leading to severe organ damage, seizures, developmental deficits, and premature death. In the United States, about 1 in 70,000 newborns is diagnosed ...