Rare diseases

News

Wave Life Sciences has lost GSK as the partner for its alpha-1 antitrypsin deficiency drug, but other partnered RNA-editing programmes remain active.

News

The FDA has turned down Pharming's bid to broaden the label of Joenja for rare immunodeficiency disorder APDS so it can be used in younger patients.

News

Sanofi has reported positive phase 3 results with venglustat in Gauder disease, but the drug missed the mark in a Fabry disease trial.

News

Boehringer's TRPC6 inhibitor apecotrep, formerly developed for COVID-19, has shown efficacy in the rare kidney disease FSGS in a phase 2 trial.

News

An EMA nod for Otsuka's Dawnzera offers EU patients with HAE a drug that can be self-administered by patients with up to eight weeks between doses.

News

Children with a devastating, rare disorder of copper metabolism, called Menkes disease, now have an FDA-approved treatment for the first time.