Forum
Carbamoylphosphate synthetase deficiency

/static/themes-v3/default/images/default/home/bg-generic.jpg?1516194360

Back

Ongoing discussions - Carbamoylphosphate synthetase deficiency

Living with diseases of nutrition, metabolism and endocrine glands

Current topics:

- US Approves First RNA Interference Drug
Alnylam’s Onpattro (patisiran) can treat a rare hereditary disease by silencing a specific gene. The first therapy based on RNA interference (RNAi) gene silencing has been approved by the US Food a

See all the discussions

News and information

Fact sheet

Carbamoylphosphate synthetase deficiency

Newsfeed

US Approves First RNA Interference Drug