Rebecca Sanders from Lipodystrophy UK tells us how the patient voice helped convince NICE to approve a much-needed drug for this rare disease, and explores how regulators and pharma compani
The mainstay of therapy for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) is Alexion's infusion therapy Soliris, but Novartis' iptacopan could provide an oral option afte
Sanofi's hopes of a speedy approval of first-in-class C1s inhibitor sutimlimab in rare disorder cold agglutinin disease (CAD) were dashed by the FDA, but new phase 3 data keep the drugmaker
Shortly after failing a Parkinson's disease study, Sanofi's venglustat has missed the mark in another trial in autosomal dominant polycystic kidney disease (ADPKD), whittling down the list
US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift.
Apellis Pharma has secured FDA approval for its complement C3 inhibitor Empaveli as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) – with a label that will allow it to challenge
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.