Rare diseases

News

AstraZeneca's long-acting Strensiq follow-up efzimfotase alfa has hit the mark in children with rare disease HPP, but the data in adults is unclear.

News

Rocket Pharma has won FDA approval for the first gene therapy for LAD-I, a devastating genetic disorder causing severe vulnerability to infections.

News

Denali's Avlayah is the first drug for neurological complications associated Hunter syndrome to be approved in the US.

News

Rhythm Pharma has secured the first approval in the US for a drug to treat hypothalamic obesity.

News

Alfasigma has claimed the first approval, in the US, for linerixibat, a drug for liver disease PBC licensed from GSK for $690m a few days ago.

News

BioMarin has suspended dosing in phase 2 trials of its achondroplasia therapy Voxzogo aimed at broadening the drug's label.