LAS VEGAS, Feb. 24, 2021 /PRNewswire/ -- Rare diseases approximately affect 300 million people globally. In the US, a rare disease affects fewer t ...

The research, published in Science Translational Medicine, found that messenger RNA (mRNA) could be used to correct a rare liver genetic disease known as argininosuccinic aciduria in a mouse model ...

Argininosuccinic aciduria is an inherited metabolic disorder that affects how the body breaks down protein—potentially leading to high levels of ammonia in the blood.

Argininosuccinate lyase deficiency (ASLD), also known as argininosuccinic aciduria, is a disease that has been enriched in the Finnish genetic heritage. In this severe metabolic disease, the body ...

Argininosuccinate lyase deficiency (ASLD), also known as argininosuccinic aciduria, is a disease that has been enriched in the Finnish genetic heritage.

People with the urea cycle disorder argininosuccinic aciduria, caused by a deficiency of ASL, have systemic NO deficiency, which can be rescued by the use of an alternative, NOS-independent ...

The research, published in Science Translational Medicine, found that messenger RNA (mRNA) could be used to correct a rare liver genetic disease known as argininosuccinic aciduria in a mouse model of ...

Argininosuccinic aciduria is an inherited metabolic disorder that affects how the body breaks down protein – potentially leading to high levels of ammonia in the blood.