UK-based biotech ReNeuron has announced encouraging results from an early stage trial of its cell therapy for the rare blindness-causing disease, retinitis pigmentosa (RP).
UK health technology startup Mendelian has received a £500,000 grant from a government agency to build products that will help GPs identify patients with rare or hard to diagnose conditions
Catalyst Pharmaceuticals has defended hiking the price of the rare disease drug Firdapse (amifampridine) from virtually nothing to more than $300,000 per year following criticism from Senat
The US regulator has approved Novo Nordisk’s long-acting therapy for haemophilia A, Esperoct, as the Danish pharma seeks to maintain its presence in an increasingly competitive sector of th
The controversy over rising drug prices in the US has been reignited after it emerged that the yearly cost of the life-sustaining rare disease drug Firdapse has been hiked from free to more
Vertex has revealed financial figures that are likely to go down well with investors, but not patients in the UK who are eligible to receive the revolutionary drugs like Orkambi yet are bei