Scientists at St. Jude Children's Research Hospital have shown that a class of drugs they pioneered has the potential to treat a rare metabolic disorder called propionic acidemia. The findings were ...

Propionic acidemia is a rare and life-threatening disorder in which mutations in genes encoding the enzyme PCC (involved in propionate metabolism) lead to a defective protein and severe metabolic ...

The Institute for Quality and Efficiency in Health Care (IQWiG) was commissioned by the Federal Joint Committee (G-BA) to investigate whether screening for vitamin B12 deficiency and the diseases ...

Moderna announces open IND for Propionic Acidemia program (mRNA-3927) Tweet this “We are excited to have a second open IND for a rare disease program and are preparing to move mRNA-3927 into a Phase 1 ...

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 ...

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 / ...

Metabolic cardiomyopathy is an important diagnostic consideration in a young patient with unexplained ventricular dysfunction or sudden cardiac arrest. Propionic acidemia is an uncommon but important ...