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 ...

Moderna to receive up to $160 million in upfront and near-term development and regulatory milestones, in addition to commercial and sales milestones and tiered royalties on net sales CAMBRIDGE, MA / ...

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 ...

Cholesterol bloodstream The FDA has granted Fast Track designation to mRNA-3927 (Moderna), an investigational mRNA therapeutic for propionic acidemia. The Food and Drug Administration (FDA) has ...

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 ...

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 ...

CAMBRIDGE, MA / ACCESSWIRE / April 3, 2024 / Moderna, Inc. (NASDAQ:MRNA) today announced that interim data for a first-in-human, phase 1/2, open-label, dose optimization study and extension study, ...

CAMBRIDGE, MA / ACCESSWIRE / May 19, 2023 / Moderna,Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported on interim data from the Phase 1/2 ...

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 ...

"Propionic acidemia is a serious rare disease with a significant unmet medical need due to the lack of disease modifying treatment options to date. We look forward to partnering with Moderna," said ...