Onlymyhealth

What Is Propionic Acidemia? The Rare Disorder That Forced a Two-Year-Old to Undergo a Liver Transplant

Propionic Acidemia is a rare genetic disorder where the body cannot process certain fats and proteins This causes toxic acid buildup in the liver which can cause life-threatening complications ...
Medical Xpress

Research reveals new treatment strategy for propionic acidemia

Propionic acidemia is caused by mutations in an enzyme called propionyl-CoA carboxylase. CoA is a helper molecule essential for energy metabolism. Propionic acidemia is typically managed by diet. The ...
Monthly Prescribing Reference

Carbaglu Approved for Hyperammonemia in Propionic Acidemia, Methylmalonic Acidemia

The Food and Drug Administration (FDA) has approved a new indication for Carbaglu ® (carglumic acid; Recordati Rare Diseases) for use as adjunctive therapy in the treatment of acute hyperammonemia due ...
Business Insider

RECORDATI ANNOUNCES STRATEGIC COLLABORATION WITH MODERNA TO DEVELOP AND COMMERCIALIZE WORLDWIDE mRNA 3927 FOR THE TREATMENT OF PROPIONIC ACIDEMIA

mRNA-3927 is a strong strategic fit with Recordati’s rare Metabolic portfolio Collaboration combines Moderna’s expertise in mRNA technology for rare metabolic disorders with Recordati’s established ...
Nasdaq

Moderna Receives FDA Fast Track Designation for Propionic Acidemia Program (mRNA-3927)

mRNA-3927 uses the same proprietary LNP formulation as mRNA-1944 (antibody against Chikungunya virus) and mRNA-3704 (methylmalonic acidemia) “Fast Track designation underscores the urgent need for a ...