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
NHS patients in Scotland with the rare disease atypical haemolytic uremic syndrome (aHUS) will now be able to access treatment with Alexion's Ultomiris, ahead of their counterparts in Engla
UK biotech Silence Therapeutics has launched an online game to raise the profile of the rare blood disorder thalassaemia, and one of its most common symptoms.
Calliditas Therapeutics could have its first product on the market in the US this autumn, as the FDA has now started a priority review of Nefecon for rare disease primary IgA nephropathy (I