Rare diseases

R&D

There are some 7,000 known rare diseases in the world, yet, multiple challenges and unmet needs remain when it comes to therapies for these diseases.

News

Ionis Pharma's Tryngolza (olezarsen) has become the first FDA-approved drug therapy for adults with rare disease familial chylomicronaemia syndrome.

News

Neurocrine Bioscience's Crenessity is the first new option for classic congenital adrenal hyperplasia (CAH) in more than 70 years.

News

Novo Nordisk has started building its first brand-new manufacturing facility in Denmark this century and will spend around $1.2bn on the plant.

News

Readout from NEXUS study sets up mid-2025 refiling for Minoryx and Neuraxpharm's leriglitazone for rare disease cerebral adrenoleukodystrophy (cALD).

News

Applied Therapeutics gets a warning letter from the FDA over a trial of rare disease drug govorestat, which was rejected by the agency last week.