Rare diseases

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.

News

Trials of AstraZeneca's anselamimab and Regeneron's Lynozyfic could deliver much-needed new therapeutic options for rare disease AL amyloidosis.