Rare diseases

News

Shionogi has reached a $2bn agreement to buy Anglo-US biotech IntraBio and its recently approved Aqneursa therapy for two rare genetic disorders.

News

CSL has licensed rights to a potentially first-in-class anti-claudin-1 antibody developed by Alentis in a deal that could be worth up to $1.55bn.

News

Patients in England and Wales with familial chylomicronaemia syndrome will soon have a new treatment option, as NICE backs Sobi's Tryngolza.

News

Sweden's Egetis has won approval in the US for the first-ever treatment for people living with the ultra-rare genetic disease MCT8 deficiency.

News

A new study suggests long-read 'nanopore' sequencing could transform the diagnosis of inherited diseases and unlock new insights for drug discovery.

News

Mirum's new oral therapy for rare bone disease fibrodysplasia ossificans progressiva (FOP) looks set to challenge Regeneron's intravenous Pasatru.