The US biotech has presented interesting preliminary data from the first four patients treated in the Phase I/II STAAR study evaluating isaralgagene civaparvovec for Fabry.

A mix of equity and debt financing has raised enough to satisfy Santhera's liquidity needs until mid-2022, which will be after the Swiss firm has filed its Duchenne drug vamorolone.

Close to 90% of the FDA’s 2021 first half novel approvals were cleared in the US before Europe, while the EU’s EMA acted first on only 15% of its first half marketing authorizations for new active substances. Exclusive Pink Sheet analysis explores use of expedited review schemes, special pathways and other comparative trends.

Jean-Paul Clozel, whose firm Actelion was bought by Johnson & Johnson for $30bn, is back with his new company and a new approach to help save the lives of heart attack patients.