Rare diseases

News

Armed with new phase 3 data, Sanofi will try to expand the US label of its Pompe disease drug Nexviazyme to include the severe infantile-onset form.

News

Johnson & Johnson's 'Swiss army knife' drug Imaavy has shown efficacy in a pivotal trial involving patients with rare autoimmune disorder wAIHA.

News

Novartis' $12bn takeover of Avidity could potentially give it the first therapy for genetic disorder facioscapulohumeral muscular dystrophy.

News

Sanofi's neurology pipeline has had a hole blown in it after a phase 3 trial of riliprubart in rare disease CIDP was stopped for futility.

News

The FDA has laid out a framework to use 'prior knowledge' on manufacturing, non-clinical, and clinical data to support cell and gene therapy filings.

News

The SARCO41 study suggests Lilly's Verzenio could become the first targeted therapy for rare, aggressive tumour dedifferentiated liposarcoma.