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It results in unhealthy levels of products otherwise normally present in the body. Defects in different enzymes lead to different types of organic aciduria. For example, propionic acidemia is another ...
mRNA-3927 is under development for the treatment of propionic acidemia in pediatrics. It is developed based on messenger RNA expression and N2GL formulation platform. The therapeutic candidate ...
Sandra A. Banta-Wright, MN, RNC, NNP; Robert D. Steiner, MD Neonates with profound and prolonged hyperammonemia with coma due to urea cycle defect will have had a neurological insult to the brain ...
Members of the Philippine Health Insurance Corp. (PhilHealth) will soon enjoy enhanced benefit packages, which include coverage for rare diseases, expected to be available in early 2025. ICYMI, the ...
A proposed latent vaccine market TAM of up to $10bn per annum may have to be parked altogether, and rare disease — Moderna is targeting Propionic Acidemia and methylmalonic acidemia (MMA ...
In this category, the drugs most advanced in clinical studies are propionic acidemia (PA) and methylmalonic acidemia (MMA). For propionic acidemia (PA), the company is evaluating the efficacy of ...
Metabolic disorders occur when the body's usual metabolic processes are disrupted. These diseases can be congenital or acquired, for example, diabetes and phenylketonuria. Partitioning clustering ...