Rare diseases

News

Chiesi has boosted its rare disease business with an agreement to buy KalVista and Ekterly, its oral drug for treating attacks in rare disease HAE.

News

Intellia has reported the first-ever phase 3 results with an in vivo gene-editing therapy, CRISPR-based lonvo-z for hereditary angioedema.

News

Novo Nordisk has claimed a key win in its head-to-head race with Agios to bring an oral PKR activator to the market for sickle cell disease.

News

A scheme to hasten the diagnosis of children with genetic disorders is achieving strong results and could be a template for national health systems.

News

Shrugging off an earlier phase 3 disappointment, Travere’s Filspari has become the first FDA-approved treatment for rare kidney disease FSGS.

News

Neurocrine Biosciences has agreed a $2.9bn deal to buy Soleno Therapeutics and its treatment for a rare, genetic form of severe obesity.