“Fast Track designation underscores the urgent need for a therapy that treats the underlying cause of propionic acidemia,” said Tal Zaks, M.D., Ph.D., chief medical officer at Moderna. “We ...

About Propionic Acidemia Propionic acidemia is a rare, serious, inherited metabolic disorder with significant morbidity and mortality, affecting one in 100,000-150,000 individuals worldwide.

Recordati Rare Diseases: Carbaglu® (carglumic acid) tablets 200mg receives U.S. FDA approval for a new indication to treat acute hyperammonemia associated with propionic acidemia and ...

Since there were almost no research programs then, Chertow and four other families launched the Propionic Acidemia Foundation in 2002 to accelerate research. The organization has funded about $1.5 ...

Propionic acidemia is an uncommon but important cause of metabolic cardiomyopathy because of an enzyme defect that impairs the metabolism of proteins and fats. Dietary nonadherence in patients with ...

Propionic acidemia (PA) and methylmalonic acidemia (MMA) are rare inherited metabolic disorders. PA results from a deficiency of the enzyme propionyl-CoA carboxylase.

Lucy Gavin’s son, Toby, was born eight weeks ago and the family quickly realised that something was not quite right with him.

The propionic acidemia market size has grown steadily in recent years. It will grow from $2.00 billion in 2024 to $2.09 billion in 2025 at a compound annual growth rate CAGR of 4.8%.

Creating awareness for a little known disease — propionic acidemia — was important to students who raised money during the annual Deerfield High School School Chest drive this year because the ...