Rare diseases

News

Biogen has started a new phase 3 trial that aims to extend the use of its Friedreich's ataxia drug Skyclarys to patients as young as two.

News

People in England and Wales with severe skin disease GPP will soon be able to access a new therapy currently denied to patients in Scotland.

News

CSL has won FDA approval for Andembry, its once-monthly subcutaneous drug to prevent hereditary angioedema (HAE) attacks.

News

Sanofi has bolstered its rare disease business with an agreement to buy Blueprint Medicines and blockbuster-in-waiting Ayvakit for ASM.

News

Stealth BioTherapeutics' filing for Barth syndrome therapy elamipretide has been turned down by the FDA, but there may be a way forward for the drug.

News

Savara shares are pummelled after the FDA says it cannot accept a filing for lead drug Molbreevi, but it is still hopeful of an approval in 2026.