Rare diseases

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.

News

The FDA has raised the pressure on Amgen to withdraw vasculitis drug Tavneos from the market with a report linking serious side effects to the drug.

News

The EU has approved UCB's Kygevvi for rare disease TK2d, and Sanofi's acute GvHD therapy Rezurock, in a pair of decisions announced this morning.